ADVERUM BIOTECHNOLOGIES, INC., 10-K filed on 4/15/2025
Annual Report
v3.25.1
Cover Page - USD ($)
$ in Millions
12 Months Ended
Dec. 31, 2024
Apr. 04, 2025
Jun. 30, 2024
Cover [Abstract]      
Document Type 10-K    
Document Annual Report true    
Document Period End Date Dec. 31, 2024    
Current Fiscal Year End Date --12-31    
Document Transition Report false    
Entity File Number 001-36579    
Entity Registrant Name Adverum Biotechnologies, Inc.    
Entity Incorporation, State or Country Code DE    
Entity Tax Identification Number 20-5258327    
Entity Address, Address Line One 100 Cardinal Way    
Entity Address, City or Town Redwood City    
Entity Address, State or Province CA    
Entity Address, Postal Zip Code 94063    
City Area Code 650    
Local Phone Number 656-9323    
Title of 12(b) Security Common Stock, par value $0.0001 per share    
Trading Symbol ADVM    
Security Exchange Name NASDAQ    
Entity Well-known Seasoned Issuer No    
Entity Voluntary Filers No    
Entity Current Reporting Status Yes    
Entity Interactive Data Current Yes    
Entity Filer Category Non-accelerated Filer    
Entity Small Business true    
Entity Emerging Growth Company false    
Document Financial Statement Error Correction true    
Document Financial Statement Restatement Recovery Analysis [Flag] true    
ICFR Auditor Attestation Flag false    
Entity Shell Company false    
Entity Public Float     $ 136.7
Entity Common Stock, Shares Outstanding   20,890,540  
Documents Incorporated By Reference Portions of the definitive proxy statement (the Proxy Statement) for the 2025 Annual Meeting of Stockholders of the registrant are incorporated by reference into Part III of this Annual Report on Form 10-K. If the Proxy Statement is not filed by April 30, 2025, then the registrant will file an amendment to this Form 10-K on Form 10-K/A to include the Part III information in this Form 10-K.    
Amendment Flag false    
Document Fiscal Year Focus 2024    
Document Fiscal Period Focus FY    
Entity Central Index Key 0001501756    
v3.25.1
Audit Information
12 Months Ended
Dec. 31, 2024
Audit Information [Abstract]  
Auditor Name Ernst & Young LLP
Auditor Firm ID 42
Auditor Location San Francisco, California
v3.25.1
CONSOLIDATED BALANCE SHEETS - USD ($)
shares in Thousands, $ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Current assets:    
Cash and cash equivalents $ 60,652 $ 75,000
Short-term investments 65,039 21,526
Prepaid expenses and other current assets 5,609 6,247
Total current assets 131,300 102,773
Operating lease right-of-use assets 33,611 35,024
Property and equipment, net 11,607 14,764
Restricted cash 1,976 1,976
Deposit and other long-term assets 1,347 1,231
Total assets 179,841 155,768
Current liabilities:    
Accounts payable 1,610 1,921
Accrued expenses and other current liabilities 15,620 12,584
Lease liability, current portion 5,668 5,476
Total current liabilities 22,898 19,981
Long-term liabilities:    
Lease liability, net of current portion 86,037 68,565
Other non-current liabilities 192 0
Total liabilities 109,127 88,546
Commitments and contingencies (Note 8)
Stockholders’ equity:    
Preferred stock, $0.0001 par value, 5,000 shares authorized; no shares issued and outstanding 0 0
Common stock, $0.0001 par value, 300,000 shares authorized at December 31, 2024: 20,848 and 10,143 shares issued and outstanding at December 31, 2024 and 2023, respectively 2 1
Additional paid-in capital 1,138,070 1,003,718
Accumulated other comprehensive loss (407) (473)
Accumulated deficit (1,066,951) (936,024)
Total stockholders’ equity 70,714 67,222
Total liabilities and stockholders’ equity $ 179,841 $ 155,768
Common stock, shares issued (in shares) 20,848 10,143
Common stock, shares outstanding (in shares) 20,848 10,143
Common stock, shares authorized (in shares) 300,000 300,000
Common stock, par value (in USD per share) $ 0.0001 $ 0.0001
v3.25.1
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares
Dec. 31, 2024
Dec. 31, 2023
Statement of Financial Position [Abstract]    
Preferred stock, par value (in USD per share) $ 0.0001 $ 0.0001
Preferred stock, shares authorized (in shares) 5,000,000 5,000,000
Preferred stock, shares issued (in shares) 0 0
Preferred stock, shares outstanding (in shares) 0 0
Common stock, par value (in USD per share) $ 0.0001 $ 0.0001
Common stock, shares authorized (in shares) 300,000,000 300,000,000
Common stock, shares issued (in shares) 20,848,000 10,143,000
Common stock, shares outstanding (in shares) 20,848,000 10,143,000
v3.25.1
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($)
shares in Thousands, $ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Income Statement [Abstract]                        
License revenue $ 1,000     $ 0 $ 0 $ 3,600   $ 3,600 $ 1,000 $ 3,600 $ 1,000 $ 3,600
Operating expenses:                        
Research and development 20,439 $ 17,097 $ 15,410 20,740 20,599 20,869 $ 32,507 41,468 52,946 62,208 77,041 77,486
General and administrative 14,999 15,807 13,789 14,319 13,481 15,586 29,596 29,067 44,595 43,386 63,118 55,056
Total operating expenses 35,438 32,904 29,199 35,059 34,080 36,455 62,103 70,535 97,541 105,594 140,159 132,542
Operating loss (34,438) (32,904) (29,199) (35,059) (34,080) (32,855) (62,103) (66,935) (96,541) (101,994) (139,159) (128,942)
Other income, net 2,087 2,406 2,052 1,661 1,576 1,200 4,458 2,776 6,545 4,437 8,232 5,748
Net loss before income taxes       (33,398) (32,504) (31,655)   (64,159)   (97,557) (130,927) (123,194)
Income tax benefit       (17) (21) (17)   (38)   (55) 0 1,078
Net loss (32,351) (30,498) (27,147) (33,415) (32,525) (31,672) (57,645) (64,197) (89,996) (97,612) (130,927) (122,116)
Other comprehensive gain (loss):                        
Net unrealized gain on marketable securities 241 (2) (41) 66 198 739 (43) 937 198 1,003 105 1,057
Foreign currency translation adjustment 16 10 (19) (12) (5) (7) (9) (12) 7 (24) (39) 1
Comprehensive loss $ (32,094) $ (30,490) $ (27,207) $ (33,361) $ (32,332) $ (30,940) $ (57,697) $ (63,272) $ (89,791) $ (96,633) $ (130,861) $ (121,058)
Net loss per share - basic (in USD per share) $ (1.55) $ (1.46) $ (1.65) $ (3.31) $ (3.23) $ (3.16) $ (3.08) $ (6.39) $ (4.64) $ (9.69) $ (6.62) $ (12.11)
Net loss per share - diluted (in USD per share) $ (1.55) $ (1.46) $ (1.65) $ (3.31) $ (3.23) $ (3.16) $ (3.08) $ (6.39) $ (4.64) $ (9.69) $ (6.62) $ (12.11)
Weighted-average common shares outstanding - basic (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069 19,782 10,082
Weighted-average common shares outstanding - diluted (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069 19,782 10,082
v3.25.1
CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY - USD ($)
shares in Thousands, $ in Thousands
Total
Common Stock
Additional Paid-In Capital
Accumulated Other Comprehensive Loss
Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2022   10,012      
Beginning balance at Dec. 31, 2022 $ 170,222 $ 1 $ 985,660 $ (1,531) $ (813,908)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 739        
Foreign currency translation adjustment (7)        
Net loss (31,672)        
Ending balance at Mar. 31, 2023 143,845        
Beginning balance (in shares) at Dec. 31, 2022   10,012      
Beginning balance at Dec. 31, 2022 170,222 $ 1 985,660 (1,531) (813,908)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 937        
Foreign currency translation adjustment (12)        
Net loss (64,197)        
Ending balance at Jun. 30, 2023 116,228        
Beginning balance (in shares) at Dec. 31, 2022   10,012      
Beginning balance at Dec. 31, 2022 170,222 $ 1 985,660 (1,531) (813,908)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 1,003        
Foreign currency translation adjustment (24)        
Net loss (97,612)        
Ending balance at Sep. 30, 2023 87,257        
Beginning balance (in shares) at Dec. 31, 2022   10,012      
Beginning balance at Dec. 31, 2022 170,222 $ 1 985,660 (1,531) (813,908)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Stock-based compensation expense 17,569   17,569    
Common stock issued upon exercise of stock options, shares (in shares)   0      
Common stock issued upon exercise of stock options 1   1    
Common stock issued under employee stock purchase plan (in shares)   82      
Common stock issued under employee stock purchase plan 488   488    
Common stock issued upon release of restricted stock units (in shares)   49      
Net unrealized gain on marketable securities 1,057     1,057  
Foreign currency translation adjustment 1     1  
Net loss $ (122,116)       (122,116)
Ending balance (in shares) at Dec. 31, 2023 10,143 10,143      
Ending balance at Dec. 31, 2023 $ 67,222 $ 1 1,003,718 (473) (936,024)
Beginning balance at Mar. 31, 2023 143,845        
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 198        
Foreign currency translation adjustment (5)        
Net loss (32,525)        
Ending balance at Jun. 30, 2023 116,228        
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 66        
Foreign currency translation adjustment (12)        
Net loss (33,415)        
Ending balance at Sep. 30, 2023 87,257        
Beginning balance at Dec. 31, 2023 67,222 $ 1 1,003,718 (473) (936,024)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities (41)        
Foreign currency translation adjustment (19)        
Net loss (27,147)        
Ending balance at Mar. 31, 2024 $ 163,681        
Beginning balance (in shares) at Dec. 31, 2023 10,143 10,143      
Beginning balance at Dec. 31, 2023 $ 67,222 $ 1 1,003,718 (473) (936,024)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities (43)        
Foreign currency translation adjustment (9)        
Net loss (57,645)        
Ending balance at Jun. 30, 2024 $ 137,400        
Beginning balance (in shares) at Dec. 31, 2023 10,143 10,143      
Beginning balance at Dec. 31, 2023 $ 67,222 $ 1 1,003,718 (473) (936,024)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 198        
Foreign currency translation adjustment 7        
Net loss (89,996)        
Ending balance at Sep. 30, 2024 $ 108,270        
Beginning balance (in shares) at Dec. 31, 2023 10,143 10,143      
Beginning balance at Dec. 31, 2023 $ 67,222 $ 1 1,003,718 (473) (936,024)
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Stock-based compensation expense 14,350   14,350    
Issuance of common stock, net of issuance costs of $9 (in shares)   10,573      
Stock Issued During Period, Value, New Issues $ 119,361 $ 1 119,360    
Common stock issued upon exercise of stock options, shares (in shares) 15 15      
Common stock issued upon exercise of stock options $ 146   146    
Common stock issued under employee stock purchase plan (in shares)   88      
Common stock issued under employee stock purchase plan 496   496    
Common stock issued upon release of restricted stock units (in shares)   29      
Net unrealized gain on marketable securities 105     105  
Foreign currency translation adjustment (39)     (39)  
Net loss $ (130,927)       (130,927)
Ending balance (in shares) at Dec. 31, 2024 20,848 20,848      
Ending balance at Dec. 31, 2024 $ 70,714 $ 2 $ 1,138,070 $ (407) $ (1,066,951)
Beginning balance at Mar. 31, 2024 163,681        
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities (2)        
Foreign currency translation adjustment 10        
Net loss (30,498)        
Ending balance at Jun. 30, 2024 137,400        
Increase (Decrease) in Stockholders' Equity [Roll Forward]          
Net unrealized gain on marketable securities 241        
Foreign currency translation adjustment 16        
Net loss (32,351)        
Ending balance at Sep. 30, 2024 $ 108,270        
v3.25.1
CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY (Parenthetical)
12 Months Ended
Dec. 31, 2024
USD ($)
Statement of Stockholders' Equity [Abstract]  
Issuance costs $ 8,449,000
v3.25.1
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)
$ in Thousands
3 Months Ended 12 Months Ended
Mar. 31, 2023
Dec. 31, 2024
Dec. 31, 2023
Cash flows from operating activities:      
Net loss $ (31,672) $ (130,927) $ (122,116)
Adjustments to reconcile net loss to net cash used in operating activities:      
Depreciation and amortization   3,653 5,644
Stock-based compensation expense   14,350 17,569
Net accretion of discount on marketable securities   (1,907) (1,935)
Non-cash lease expense 2,666 7,495 18,257
Non-cash sublease loss/(income) (1,567) 14,000 (89)
Loss on disposal of property and equipment   2 50
Other   (39) 225
Changes in operating assets and liabilities:      
Prepaid expenses and other current assets   638 298
Deposit and other long-term assets   (68) 182
Accounts payable   (262) (369)
Accrued expenses and other liabilities (939) 3,021 (5,396)
Lease liability   (2,418) (3,222)
Net cash used in operating activities   (92,462) (90,902)
Cash flows from investing activities:      
Purchases of marketable securities   (101,901) (36,718)
Maturities of marketable securities   60,400 134,401
Purchases of property and equipment   (388) (808)
Net cash (used in) provided by investing activities   (41,889) 96,875
Cash flows from financing activities:      
Proceeds from issuance of common stock and pre-funded warrants in private placement, net of issuance costs   119,361 0
Payment of deferred offering costs   0 (420)
Proceeds from issuance of common stock pursuant to option exercises   146 1
Proceeds from employee stock purchase plan   496 488
Net cash provided by financing activities   120,003 69
Net (decrease) increase in cash, cash equivalents and restricted cash   (14,348) 6,042
Cash, cash equivalents and restricted cash at beginning of period 70,934 76,976 70,934
Cash, cash equivalents and restricted cash at end of period   62,628 76,976
Cash and cash equivalents 67,552 60,652 75,000
Restricted cash 2,503 1,976 1,976
Cash, cash equivalents and restricted cash at end of period   62,628 76,976
Supplemental schedule of noncash information      
Non-cash settlement of operating lease liability $ 14,900 0 14,903
Remeasurement of operating lease right-of-use assets and liabilities   0 7,632
Fixed assets in accounts payable and current liabilities   $ 201 $ 91
v3.25.1
Description of the Business
12 Months Ended
Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
Description of the Business Description of the Business
Nature of the Business—Adverum Biotechnologies, Inc. (the “Company” or “Adverum”) was incorporated in Delaware on July 17, 2006 and is headquartered in Redwood City, California. The Company aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases. The Company develops gene therapy product candidates intended to provide durable efficacy by inducing sustained expression of a therapeutic protein.
Going Concern, Liquidity, Risks and Uncertainties—As of December 31, 2024, the Company has devoted substantially all of its efforts to product development and has not realized product sales revenues from its planned principal operations. The Company has a limited operating history, and the sales and income potential of the Company’s business and market are unproven. The Company has experienced net losses since its inception and, as of December 31, 2024, had an accumulated deficit of $1.1 billion. The Company used $92.5 million of cash in operations during the year ended December 31, 2024. The Company expects to continue to incur net losses and operating cash outflows for at least the next several years. A successful transition to attaining profitable operations is dependent upon achieving a level of revenue adequate to support the Company’s cost structure.
As of December 31, 2024, the Company had cash, cash equivalents and short-term investments of $125.7 million, which are expected to fund the Company’s operations into the second half of 2025. The Company has determined that its cash and cash equivalents as of December 31, 2024 would be insufficient to fund its operations for a period of at least twelve months from the date these financial statements are issued, which raises substantial doubt regarding the Company’s ability to continue as a going concern.
The Company’s financial statements have been prepared using the going concern basis of accounting, which contemplates the continuity of operations, realization of assets and the satisfaction of liabilities and commitments in the ordinary course of business. The financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might result from the outcome of the uncertainties described above.
Management expects operating losses to continue for the foreseeable future. The Company plans to raise substantial additional capital to continue as a going concern, including through public or private equity or debt financings, third-party funding, revenue interest arrangements, collaborations, strategic alliances and licensing arrangements, or any combination of these approaches. The Company may be unable to raise additional funds or to enter into such agreements or arrangements on favorable terms, or at all. If the Company is unable to raise capital or enter into such agreements as and when needed, it may have to delay, limit, reduce or terminate its product development programs, commercialization efforts or other operations, or to cease operations or liquidate its assets. Although the Company has been successful in raising capital in the past, there is no assurance that it will be successful in obtaining such additional financing. Therefore, it is not considered probable that the Company’s plans to raise additional capital will alleviate the substantial doubt regarding its ability to continue as a going concern.
v3.25.1
Restatement of Previously Issued Financial Statements
12 Months Ended
Dec. 31, 2024
Accounting Changes and Error Corrections [Abstract]  
Restatement of Previously Issued Financial Statements Restatement of Previously Issued Financial Statements
The Company has restated herein its audited consolidated financial statements for the year ended December 31, 2023 in accordance with Accounting Standards Codification (“ASC”) Topic 250, Accounting Changes and Error Corrections. In March 2025, the Company discovered that there were errors in its lease accounting related to tenant improvement allowances in connection with an operating lease agreement for a building in North Carolina, which the Company entered into in January 2021 and subsequently subleased in October 2021 (the operating lease agreement and sublease agreements, with their respective amendments, collectively, the “NC Leases”). The Company inappropriately omitted accounting for available tenant improvement allowance that resulted in a misstatement of its operating lease right-of-use asset and related lease liability. Additionally, the Company did not account for tenant improvement allowances that were conveyed to the subtenant, resulting in an overstatement of sublease income. The tables below reflect the adjustments related to this restatement, the amounts of which are included with reference (a).
The Company has also restated interim period financial statements for the first, second and third quarters of 2024 and 2023 as a result of these errors. See Note 14 Quarterly Financial Information (Unaudited) for additional information on the restated interim financial statements for the first, second, and third quarters of 2024 and 2023.
As part of this restatement, the Company has corrected certain other immaterial errors. While these other errors are quantitatively and qualitatively immaterial, individually and in the aggregate, because the Company is restating its consolidated financial statements for material errors, the Company has corrected other immaterial errors as well. These adjustments are described in more detail in references (b) and (c) and elsewhere in the section titled “Other Adjustments” below.
Other Adjustments
The adjustments to correct other errors that are quantitatively and qualitatively immaterial, individually and in the aggregate are as follows:
(b)The Company had previously recorded an out of period adjustment related to research and development expenses in the first quarter of 2023 that was the result of not recording a severance accrual of $0.2 million in 2022.
(c)The Company had previously understated its right-of-use assets and lease liability by $0.3 million related to its facilities in Redwood City, California at each balance sheet in 2024 and 2023.
The Company also identified certain errors as a result of imprecise calculations related to its research and development tax credits that should have been corrected in the consolidated financial statements for the year ended December 31, 2023. Such amounts are deemed immaterial to the Company’s income tax provision for the years ended December 31, 2024 and 2023.
The remainder of the notes to the Company’s financial statements have been restated, as applicable, to reflect the impacts from the restatement discussed above.
Impact of the Restatement
The following tables represent the restated amounts in the Consolidated Balance Sheet, Statement of Operations and Comprehensive Loss, and Statement of Cash Flows for the year ended December 31, 2023 (in thousands, except per share data).
The amounts as previously reported for fiscal year 2023 were derived from the Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed on March 18, 2024 and the amounts previously reported for the unaudited condensed consolidated quarterly financial information for the quarterly periods in the years ended December 31, 2023 and 2024 were derived from the Company’s Quarterly Reports on Form 10-Q for the quarterly periods within those years (the “Previously Reported Amounts”). The Previously Reported Amounts are labeled as “As Reported” in the tables below. The amounts labeled “Adjustment” represent the effects of this restatement described above.
The cumulative effect of the error on periods prior to 2023 was $11.3 million which is included in the beginning balance of the accumulated deficit of the restated Consolidated Balance Sheet for the year ended December 31, 2023.
Consolidated Balance Sheet
As of December 31, 2023
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$75,000 $— $75,000 
Short-term investments21,526 — 21,526 
Prepaid expenses and other current assets6,247 6,247 
Total current assets102,773 — 102,773 
Operating lease right-of-use assets52,266 (17,242)
(a) (c)
35,024 
Property and equipment, net14,764 — 14,764 
Restricted cash1,976 — 1,976 
Deposit and other long-term assets1,231 1,231 
Total assets$173,010 $(17,242)$155,768 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable$1,921 $— $1,921 
Accrued expenses and other current liabilities12,584 — 12,584 
Lease liability, current portion10,409 (4,933)
(a)
5,476 
Total current liabilities24,914 (4,933)19,981 
Long-term liabilities:
Lease liability, net of current portion64,627 3,938 
(a) (c)
68,565 
Total liabilities89,541 (995)88,546 
Commitments and contingencies
Stockholders’ equity:
Preferred stock, $0.0001 par value, 5,000 shares authorized; no shares issued and outstanding
— — — 
Common stock, $0.0001 par value, 300,000 shares authorized at December 31, 2023: 10,143 shares issued and outstanding at December 31, 2023
— 
Additional paid-in capital1,003,718 — 1,003,718 
Accumulated other comprehensive loss(473)— (473)
Accumulated deficit(919,777)(16,247)
(a) (b)
(936,024)
Total stockholders’ equity83,469 (16,247)67,222 
Total liabilities and stockholders’ equity$173,010 $(17,242)$155,768 
Consolidated Statement of Operations and Comprehensive Loss
Year ended December 31, 2023
As Previously Reported
Adjustment
Reference
As Restated
License revenue$3,600 $— $3,600 
Operating expenses:
Research and development77,676 (190)
(b)
77,486 
General and administrative49,915 5,141 
(a)
55,056 
Total operating expenses127,591 4,951 132,542 
Operating loss(123,991)(4,951)(128,942)
Other income, net5,748 — 5,748 
Net loss before income taxes(118,243)(4,951)(123,194)
Income tax benefit
1,078 — 1,078 
Net loss$(117,165)$(4,951)$(122,116)
Other comprehensive gain:
Net unrealized gain on marketable securities
1,057 — 1,057 
Foreign currency translation adjustment— 
Comprehensive loss$(116,107)$(4,951)$(121,058)
Net loss per share - basic and diluted$(11.62)$— $(12.11)
Weighted-average common shares outstanding - basic and diluted10,082 — 10,082 
Consolidated Statement of Cash Flows
Year ended December 31, 2023
As Previously Reported
Adjustment
Reference
As Restated
Cash flows from operating activities:
Net loss$(117,165)$(4,951)
(a) (b)
$(122,116)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense13,027 5,230 
(a)
18,257 
Non-cash sublease income
— (89)(89)
Changes in operating assets and liabilities:
Accrued expenses and other liabilities(5,206)(190)
(b)
(5,396)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets and liabilities$13,711 $(6,079)
(a) (c)
$7,632 
There was no impact on net cash used in operating activities or within any line items within investing and financing activities.
v3.25.1
Summary of Significant Accounting Policies
12 Months Ended
Dec. 31, 2024
Accounting Policies [Abstract]  
Summary of Significant Accounting Policies Summary of Significant Accounting Policies
Basis of Presentation and Principles of Consolidation—The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). The consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions and balances have been eliminated in consolidation.
Use of Estimates—The preparation of financial statements in conformity with U.S. GAAP requires management to make judgements, estimates and assumptions that affect the reported amounts of assets, liabilities, revenue and expenses, and related disclosures. Management bases its estimates on historical experience and on various other assumptions that are believed to be reasonable under the circumstances. These estimates form the basis for making judgments about the carrying values of assets and liabilities when these values are not readily apparent from other sources. Accounting estimates and judgements are inherently uncertain, and the actual results could differ from these estimates.
March 2024 Reverse Stock Split—On March 21, 2024, the Company effected a 1-for-10 reverse stock split of its common stock. The par value and the authorized shares of the common stock were not adjusted as a result of the reverse stock split.
All equity related information including per share amounts for all periods presented in these consolidated financial statements and the notes thereto have been adjusted retroactively, where applicable, to reflect this reverse stock split.
Foreign Currency—Assets and liabilities of non-U.S. subsidiaries that operate in a local currency environment, where the local currency is the functional currency, are translated to U.S. dollars at exchange rates in effect at the balance sheet date, with the resulting translation adjustments directly recorded to a separate component of accumulated other comprehensive loss. Upon sale or upon complete or substantially complete liquidation of an investment in a foreign entity, the amount attributable to that entity and accumulated in the translation adjustment component of equity is removed from the separate component of equity and reported as part of the gain or loss on sale or liquidation of the investment for the period during which the sale or liquidation occurs. Income and expense accounts are translated at average exchange rates for the period. Transactions which are not in the functional currency of the entity are remeasured into the functional currency and gains or losses resulting from the remeasurement recorded in other income, net.
Cash and Cash Equivalents—The Company considers all highly liquid investments purchased with original maturities of three months or less at the date of purchase to be cash equivalents. Cash and cash equivalents include cash held in banks, money market accounts and highly liquid debt securities. Cash equivalents are stated at fair value.
Restricted Cash—Restricted cash primarily consists of cash collateral to letter of credit provided to the landlord in relation to a lease agreement. See Note 6, Leases for additional information.
Short-Term Investments—All short-term investments in debt securities have been classified as “available for sale” and are carried at fair value. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive loss and reported as a separate component of stockholders’ equity until realized. The cost of securities sold is based on the specific-identification method. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. Interest on short-term investments is included in other income, net in the Company’s consolidated statements of operations and comprehensive loss. In accordance with the Company’s investment policy, management invests to diversify credit risk and only invests in securities with high credit quality, including U.S. government securities.
The Company assesses available-for-sale debt securities on a quarterly basis to see whether any unrealized loss is due to credit-related factors. Factors considered in determining whether an impairment is credit-related include the extent to which the investment’s fair value is less than its cost basis, declines in published credit ratings, changes in interest rates, and any other adverse factors related to the security. If it is determined that a credit-related impairment exists, the Company will measure the credit loss based on a discounted cash flow model. Credit-related impairments on available-for-sale debt securities are recognized as an allowance for credit losses with a corresponding adjustment to other income, net in the Company’s consolidated statement of operations. The unrealized loss position that is not credit-related is recorded, net of any related tax effects, in other comprehensive income until realized. There were no credit-related losses recognized for the periods presented.
Accrued Interest Receivable—Accrued interest receivable related to the Company’s available-for-sale debt securities is presented within prepaid expenses and other current assets on the Company’s consolidated balance sheets. The Company has elected to exclude accrued interest receivable from both the fair value and the amortized cost basis of available-for-sale debt securities for the purposes of identifying and measuring any impairment. The Company writes off accrued interest receivable once it has determined that the asset is not realizable. Any write offs of accrued interest receivable are recorded by reversing interest income, recognizing credit loss expense, or a combination of both. To date, the Company has not written off any accrued interest receivables associated with its available-for-sale debt securities.
Segment Reporting—The Company operates and manages its business as one reporting and operating segment, which is the business of developing and commercializing gene therapeutics. The Company’s chief executive officer, who is the chief operating decision maker (“CODM”), assesses segment performance and decides how to allocate resources based on total operating expenses as reported on the consolidated statements of operations and comprehensive loss. See Note 11, Segment Reporting, for additional information.
Concentrations of Credit Risk and Other Uncertainties—Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash, cash equivalents and short-term investments. Risks associated with cash, cash equivalents, short-term investments are mitigated by the Company’s investment policy, which limits the Company’s investing to securities having specified credit ratings. Management believes that the Company is not exposed to significant credit risk.
The Company is subject to certain risks and uncertainties, including, but not limited to changes in any of the following areas that the Company believes could have a material adverse effect on future financial position or results of operations: the ability to obtain future financing; regulatory approval and market acceptance of, and reimbursement for, the Company’s product candidates; performance of third-party clinical research organizations and manufacturers; development of sales channels; protection of intellectual property; litigation or claims against the Company based on intellectual property, patent, product, regulatory or other factors; and the Company’s ability to attract and retain employees necessary to support growth.
Property and Equipment—Property and equipment are recorded at cost, net of accumulated depreciation and amortization. Depreciation is recorded using the straight-line method over the estimated useful lives of the assets, generally three to five years. Leasehold improvements are capitalized and amortized over the shorter period of their expected lives or the lease term. Major replacements and improvements are capitalized, while general repairs and maintenance are expensed as incurred.
Impairment of Long-Lived Assets—Long-lived assets, including property and equipment and finite-lived intangible assets, are reviewed for impairment whenever facts or circumstances either internally or externally may indicate that the carrying value of an asset may not be recoverable. If there is an indication of impairment, the Company tests for recoverability by comparing the estimated undiscounted future cash flows expected to result from the use of the asset to the carrying amount of the asset or asset group. If the asset or asset group is determined to be impaired, any excess of the carrying value of the asset or asset group over its estimated fair value is recognized as an impairment loss.
Leases For long-term operating leases, the Company recognizes a right-of-use asset and a lease liability on the Company's consolidated balance sheets. The Company determines if an arrangement contains a lease and the classification of the lease at inception. An arrangement contains a lease if there is an identified asset and if the Company controls the use of the identified asset throughout the period of use. The lease liability is determined as the present value of future lease payments and lease incentives over the expected lease term. Lease incentives are included in the measurement of the consideration in the contract at lease commencement when the Company is reasonably certain to incur costs to construct lessee assets. In order to determine the incremental borrowing rate, the Company determines its credit rating, adjusts the credit rating for the nature of the collateral, and benchmarks the borrowing rate against observable yields on comparable securities with a similar term. The incremental borrowing rate, the ROU asset and the lease liability are reevaluated upon a lease modification. It bases the ROU asset on the lease liability adjusted for any prepaid or deferred rent. The Company elected to combine lease and non-lease components for all underlying assets groups. The Company determines the lease term at the commencement date by considering whether renewal options and termination options are reasonably assured of exercise. For short term leases, the Company does not recognize a right-of-use asset and lease liability and recognizes the lease expense over the term of the lease on a straight-line basis.
Rent expense for operating leases is recognized on a straight-line basis over the lease term and is included in operating expenses on the statements of operations and comprehensive loss. The variable lease payments primarily consist of common area maintenance and other operating costs.
The Company assesses the probability of collecting lease payments under its sublease at each reporting date. The Company currently does not deem the collection of lease payments probable and therefore sublease income is constrained at the lesser of cash payments or straight-line sublease income. Sublease income/loss for operating leases is classified in general and administrative expenses based on payments collected and tenant improvement allowance disbursed to the subtenant.
Revenue Recognition—The Company has primarily generated revenue through license, research and collaboration arrangements with its strategic partners.
The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, Revenue from Contracts with Customers, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.
Research and Development Expenses—Research and development expenses are charged to expense as incurred. Research and development expenses include primarily personnel-related costs, stock-based compensation expense, laboratory supplies, consulting costs, external contract research and development expenses, including expenses incurred under agreements with contract research organizations (“CROs”), the cost of acquiring, developing and manufacturing clinical trial materials, and overhead expenses, such as rent, equipment depreciation, insurance and utilities. Advance payments for goods or services for future research and development activities are deferred and expensed as the goods are delivered or the related services are performed.
The Company estimates research and clinical trial expenses based on the services performed pursuant to contracts with research institutions and clinical research organizations that conduct and manage nonclinical studies and clinical trials on the Company’s behalf. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. These estimates are based on communications with the third-party service providers and the Company’s estimates of accrued expenses and on information available at each balance sheet date. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual accordingly.
Fair Value Measurements—Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The carrying amounts of the Company’s financial instruments, including cash equivalents approximate their fair values due to their short-term maturities. See Note 4, Fair Value Measurements for the methodologies and assumptions used in valuing financial instruments.
Stock-based Compensation Expense—Stock-based compensation expense related to stock awards to employees is measured at fair value of the award on the date of the grant. The Company estimates the grant-date fair value, and the resulting stock-based compensation expense, using the Black-Scholes valuation model for stock options and employee stock purchase and using intrinsic value, which is the closing price of its common stock on the date of the grant, for restricted stock units (“RSUs”) and performance stock units (“PSUs”). Expense recognition of PSU and performance-based options commences when the associated performance-based criteria are determined to be probable.
The fair value of the award that is ultimately expected to vest is recognized as expense on a straight-line basis over the requisite service period, which is generally the vesting period.
The Black-Scholes valuation model requires the use of following assumptions:
Expected Term—The expected term assumption represents the period that the Company’s stock-based awards are expected to be outstanding and is determined using the simplified method.
Expected Volatility—Expected volatility is based on the Company’s historical stock price volatility.
Expected Dividend—The Black-Scholes valuation model calls for a single expected dividend yield as an input. The Company has never paid dividends and has no plans to pay dividends.
Risk-Free Interest Rate—The risk-free interest rate is based on the U.S. Treasury zero-coupon issues in effect at the time of grant for periods corresponding with the expected term of option.
Income Taxes—The Company accounts for income taxes using the asset and liability method. The Company records deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse.
In evaluating the ability to recover its deferred income tax assets, the Company considers all available positive and negative evidence, including its operating results, ongoing tax planning and forecasts of future taxable income on a jurisdiction-by-jurisdiction basis. In the event the Company determines that it would be able to realize its deferred income tax assets in the future in excess of their net recorded amount, it would make an adjustment to the valuation allowance that would reduce the provision for income taxes. Conversely, in the event that all or part of the net deferred tax assets are determined not to be realizable in the future, an adjustment to the valuation allowance would be charged to earnings in the period when such determination is made. As of December 31, 2024 and 2023, the Company has recorded a full valuation allowance on its deferred tax assets.
Tax benefits related to uncertain tax positions are recognized when it is more likely than not that a tax position will be sustained upon examination. Interest and penalties related to unrecognized tax liabilities are included within the provision for income tax.
Comprehensive Loss—Comprehensive loss comprises net loss and other comprehensive loss. Other comprehensive loss consists of foreign currency translation adjustments and unrealized gain or loss on marketable securities.
Basic and Diluted Net Loss Per Share Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period. Diluted net loss per share is computed by giving effect to all potential dilutive common stock equivalents outstanding for the period using the treasury stock method. Outstanding stock options, RSUs, and employee stock purchase plan (“ESPP”) are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive.
Recent Accounting Pronouncements
In November 2024, the FASB issued ASU 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (“ASU 2024-03”). ASU 2024-03 requires disaggregated disclosures of certain categories of expenses in the notes to the financial statements that are included in expense line items on the face of the income statement in annual and interim periods. ASU 2024-03 is effective for annual periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027. The Company is evaluating the impact of this guidance on its financial statements and related disclosures.
In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures (“ASU 2023-09”). ASU 2023-09 requires enhanced annual disclosures regarding the rate reconciliation and income taxes paid. ASU 2023-09 is effective for annual periods beginning after December 15, 2024 and may be adopted on a prospective or retrospective basis. Early adoption is permitted. The Company is evaluating the impact of this guidance on its financial statements and related disclosures.
In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures (“ASU 2023-07”). The amendments in ASU 2023-07 are intended to improve reportable segment disclosure, primarily through enhanced disclosures about significant segment expenses. ASU 2023-07 is effective for annual periods beginning after December 15, 2023, and interim periods beginning after December 15, 2024. The amendments in this ASU should be applied retrospectively to all prior periods presented in the financial statements. The Company retrospectively adopted ASU 2023-07 on December 31, 2024. The adoption of ASU 2023-07 did not have a material financial impact on the Company’s consolidated financial statements.
v3.25.1
Fair Value Measurements and Fair Value of Financial Instruments
12 Months Ended
Dec. 31, 2024
Fair Value Disclosures [Abstract]  
Fair Value Measurements and Fair Value of Financial Instruments Fair Value Measurements and Fair Value of Financial Instruments
The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:
Level 1: Quoted prices in active markets for identical assets or liabilities.
Level 2: Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities, quoted prices in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities.
Level 3: Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities.
Assets and liabilities measured at fair value are classified in their entirety based on the lowest level of input that is significant to the fair value measurement. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires management to make judgments and consider factors specific to the asset or liability.
The fair value of Level 1 securities is determined using quoted prices in active markets for identical assets. Level 1 securities consist of highly liquid money market funds. Financial assets and liabilities are considered Level 2 when their fair values are determined using inputs that are observable in the market or can be derived principally from or corroborated by observable market data such as pricing for similar securities, recently executed transactions, cash flow models with yield curves, and benchmark securities. In addition, Level 2 financial instruments are valued using comparisons to like-kind financial instruments and models that use readily observable market data as their basis. U.S. government and agency securities, commercial paper and corporate bonds are valued primarily using market prices of comparable securities, bid/ask quotes, interest rate yields and prepayment spreads and are included in Level 2.
The following is a summary of the Company’s cash equivalents and short-term investments (in thousands):
December 31, 2024
Amortized
Cost Basis
Unrealized
Gains
Unrealized
Losses
Estimated
Fair Value
Level 1
Money market funds$165 $— $— $165 
Level 2
Commercial paper78,500 10 (9)78,501 
U.S. government and agency securities41,210 56 — 41,266 
Total cash equivalents and short-term investments119,875 66 (9)119,932 
Less: Cash equivalents(54,901)— (54,893)
Total short-term investments$64,974 $66 $(1)$65,039 
December 31, 2023
Amortized
Cost Basis
Unrealized
Gains
Unrealized
Losses
Estimated
Fair Value
Level 1
Money market funds$10,204 $— $— $10,204 
Level 2
Commercial paper64,693 — (35)64,658 
U.S. government and agency securities17,616 (17)17,603 
Total cash equivalents and short-term investments92,513 (52)92,465 
Less: Cash equivalents(70,972)— 33 (70,939)
Total short-term investments$21,541 $$(19)$21,526 
As the Company may sell these securities at any time for use in current operations even if the securities have not yet reached maturity, all marketable securities are classified as current assets in the Company’s consolidated balance sheets. As of December 31, 2024, all marketable securities had a remaining maturity of less than one year. The Company held 14 debt securities in an unrealized loss position with an aggregate fair value at December 31, 2024 of $54.2 million. These are highly liquid funds with high credit ratings with final maturity of less than one year from the balance sheet date. There were no individual securities that were in a significant unrealized loss position as of December 31, 2024 and 2023. The Company has not recorded an allowance for credit losses as of December 31, 2024 or 2023 related to these securities. The accrued interest receivable on available-for-sale marketable securities was immaterial at December 31, 2024 or 2023. The Company regularly reviews the securities in an unrealized loss position and evaluates the current expected credit loss by considering factors such as historical experience, market data, issuer-specific factors, and current economic conditions. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost bases. The Company has not recorded any impairment charges on available-for-sale securities.
v3.25.1
Revenue
12 Months Ended
Dec. 31, 2024
Revenue from Contract with Customer [Abstract]  
Revenue Revenue
Lexeo
On January 25, 2021, the Company and Lexeo Therapeutics, Inc (“Lexeo”) entered into a License Agreement pursuant to which the Company granted Lexeo an exclusive, worldwide, royalty-bearing license to certain of the Company’s intellectual property to develop, manufacture, and commercialize a gene therapy product to treat cardiomyopathy due to Friedreich’s Ataxia. Upon execution of the agreement, Lexeo paid the Company a one-time, non-creditable and non-refundable upfront payment of $7.5 million.
Under the terms of the agreement, the Company is eligible to receive additional payments upon the achievement of certain milestones. Additionally, the Company will receive royalty payments on net sales subject to a cap and reductions based on patent expiry, anti-stacking, and a defined royalty floor percentage.
In February 2023, Lexeo notified the Company that it had achieved the first development milestone; accordingly, the Company received and recognized $3.5 million of license revenue during the year ended December 31, 2023.
Ray
On February 6, 2023, the Company and Ray Therapeutics, Inc (“Ray”) entered into a License Agreement pursuant to which the Company granted Ray a non-exclusive, worldwide, royalty-bearing license to certain of the Company’s intellectual property to develop, manufacture, and commercialize a gene therapy product to treat retinitis pigmentosa and the right to develop the technology to treat other ocular diseases and disorders. Under the terms of the agreement, the Company is eligible to receive development, regulatory and commercial milestone payments and royalties on net sales once commercialized.
In September 2024, Ray notified the Company that it achieved the first development milestone. Accordingly, the Company recognized $1.0 million of license revenue during the year ended December 31, 2024.
v3.25.1
Leases
12 Months Ended
Dec. 31, 2024
Leases [Abstract]  
Leases Leases
Redwood City
The Company has a lease for facilities in Redwood City, California (“Redwood City Premises”), which expires December 31, 2031, with an option to extend for a period of eight years. Related to this lease, the Company provided the landlord with a letter of credit, as amended, in the amount of $2.0 million, which is classified as restricted cash under long-term assets on the Company’s consolidated balance sheets.
Prior to September 30, 2023, the Company had two facility leases in Redwood City. In March 2023, the Company entered into an amendment to accelerate the expiration of the lease for one of the facilities in its Redwood City Premises from December 31, 2031 to September 30, 2023. Concurrently, the Company entered into an agreement for additional tenant improvement allowance towards its other Redwood City Premises. The Company accounted for this amendment as a lease modification in accordance with ASC 842-10-25-11(d). As a result of the modification the Company revalued the lease liability based on the new and remaining lease terms, which resulted in a reduction to the lease liability of $7.9 million, and recognized the remeasurement to the lease liabilities as an adjustment to the right-of-use asset. The estimated value of non-cash consideration, composed primarily of leasehold improvements and furniture and fixtures, was $14.9 million, which was fully amortized as of September 30, 2023. There was no charge recognized in the consolidated statement of operations.
North Carolina
The Company has restated amounts related to this lease. See Notes 2 and 14.
On January 8, 2021, the Company entered into an operating lease agreement for a building in North Carolina (“NC Premises”). The lease commenced in April 2021, when the Company obtained control of the NC Premises, and the lease term expires in October 2037 with two options to extend the lease term for a period of five years each.
On October 26, 2021, the Company entered into a sublease agreement with Jaguar Gene Therapy, LLC (“Jaguar”) for the NC Premises through October 2037, the remainder of the lease term, and concurrently changed the terms of the head lease. In addition, the remainder of the tenant improvement allowance under the original lease of approximately $22.7 million was transferred to Jaguar.
On April 3, 2023, the Company entered into an amendment of the lease of its NC Premises with the landlord and subtenant. Under this amendment, the parties agreed to substantially reduce the total tenant improvement allowance in exchange for lower monthly rent. The Company accounted for this amendment as a lease modification in accordance with ASC 842-10-25-11(d). The Company remeasured the lease liability, resulting in an increase in the lease liability with a corresponding increase of the right-of-use asset of $0.3 million in the quarter ended June 30, 2023. There was no charge recognized in the consolidated statement of operations.
During the year ended December 31, 2022, management reassessed the probability of collection of the lease payments from Jaguar over the remaining term of a sublease. At that time, management assessed the collectibility to be less than probable and recognized an adjustment to eliminate the deferred rent receivable as a current period adjustment during the year ended December 31, 2022. Deferred rent receivable was zero as of December 31, 2024 and 2023. In April 2023, Jaguar assigned the sublease to Advanced Medicine Partners, LLC (at that time, a wholly-owned subsidiary of Jaguar, “AMP” or the “subtenant”) as the subtenant for the NC Premises for the remainder of the lease term. Pursuant to the sublease and the notice and waiver of assignment (the “assignment agreement”), Jaguar remained obligated for AMP’s proper performance under the sublease.
In February 2025, a lien totaling $4.8 million was filed by a third–party contractor against the subtenant’s interest in the NC Premises. The Company discharged the lien in March 2025 by depositing cash in the full amount with the applicable court in order to avoid a default under the head lease. Subsequently, the subtenant and Jaguar failed to remit the March 2025 rent and subsequent rent payments and defaulted, and failed to cure such defaults, under the sublease and the assignment agreement for the NC Premises. Subsequent to December 31, 2024, the subtenant made payments totaling $1.4 million for January and February 2025 rent and drew $2.3 million from the available tenant improvement allowance, at which point the subtenant discontinued rent payments. The Company assumed responsibility for rent payments in March 2025 and as of April 7, 2025 had made payments in the total payment of $1.9 million to satisfy outstanding rent and common area management fee obligations. See Note 15 Subsequent Events.
The Company recorded sublease loss of $14.0 million and sublease income of $0.1 million for the years ended December 31, 2024 and 2023, respectively, which was recognized in general and administrative expense.
As of December 31, 2024, the weighted-average remaining lease term was 11.2 years for the Company’s leases and the weighted-average Incremental Borrowing Rate (“IBR”) was 11.6%. As of December 31, 2023, the weighted-average remaining lease term was 11.6 years for the Company’s leases and the weighted-average IBR was 11.7%.
The following table summarizes the undiscounted future non-cancellable lease payments under the lease agreements as of December 31, 2024 (in thousands):
December 31,Operating Leases
2025$12,142 
202614,892 
202715,278 
202815,677 
202916,089 
Thereafter93,087 
Total undiscounted lease payments$167,165 
Less: Imputed Interest
(75,460)
Total$91,705 
Rent expense for the years ended December 31, 2024, and 2023 was $13.3 million and $25.3 million, respectively. Included in rent expense for the years ended December 31, 2024, and 2023 were variable lease costs for utilities, parking, maintenance, and real estate taxes of $2.3 million and $2.4 million, respectively. Cash paid for amounts included in the measurement of lease liabilities was $5.8 million and $7.5 million for the twelve months ended December 31, 2024 and 2023, respectively. During the years ended December 31, 2024 and 2023, the landlord made payments to the subtenant on the Company’s behalf for tenant improvement allowances in the amount of $20.5 million and $5.4 million and the subtenant made rent payments to the landlord on the Company’s behalf in the amount of $6.5 million and $5.5 million, respectively.
v3.25.1
Balance Sheet Components
12 Months Ended
Dec. 31, 2024
Balance Sheet Related Disclosures [Abstract]  
Balance Sheet Components Balance Sheet Components
Property and Equipment, Net
Property and equipment, net consisted of the following:
December 31,
20242023
(In thousands)
Laboratory equipment$14,963 $14,638 
Leasehold improvements13,779 13,586 
Computer equipment and software901 868 
Construction in progress184 
Total property and equipment29,652 29,276 
Less accumulated depreciation and amortization(18,045)(14,512)
Property and equipment, net$11,607 $14,764 
Accrued Expenses and Other Current Liabilities
Accrued expenses and other current liabilities consist of the following:
December 31,
20242023
(In thousands)
Employee compensation$9,540 $8,040 
Accrued nonclinical, clinical and process development costs4,401 3,367 
Accrued professional fees554 351 
State income tax payable123 101 
Other1,002 725 
Total accrued expenses and other current liabilities$15,620 $12,584 
v3.25.1
Commitments and Contingencies
12 Months Ended
Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]  
Commitments and Contingencies Commitments and Contingencies
License Agreements
The Company is a party to various agreements, principally relating to licensed technology that requires future payments relating to milestones or royalties on future sales of specified products. The Company recognized license revenue of $1.0 million for the first development milestone in the Ray License Agreement and $3.5 million for the first development milestone in the Lexeo License Agreement during the years ended December 31, 2024 and 2023, respectively. See Note 5, Revenue, for additional information. Because achievement of these future milestones is not fixed and determinable, such amounts have not been included on the Company’s consolidated statements of operations and comprehensive loss.
v3.25.1
Stockholders' Equity
12 Months Ended
Dec. 31, 2024
Share-Based Payment Arrangement [Abstract]  
Stockholders' Equity Stockholders’ Equity
February 2024 Private Placements
On February 7, 2024, the Company entered into a securities purchase agreement, pursuant to which the Company sold 10.5 million shares of its common stock and, in lieu of common stock, pre-funded warrants to purchase an aggregate of 75,000 shares of common stock (the “Pre-Funded Warrants”) to certain institutional and accredited investors in a private placement. The purchase price per share was $12.00, or $11.999 per Pre-Funded Warrant, which represents the purchase price per share minus the $0.001 per share exercise price of each Pre-Funded Warrant. Such Pre-Funded Warrants can be exercised at any time and have no expiration date.
The exercise of the outstanding Pre-Funded Warrants is subject to a beneficial ownership limitation of 9.99%. The Pre-Funded Warrants were classified as a component of stockholders’ equity. As of December 31, 2024, none of the Pre-Funded Warrants had been exercised.
Concurrently, the Company also entered into a securities purchase agreement with two directors of the Company (together with the private placement to certain institutional and accredited investors, the “Private Placements”). The Company issued and sold 23,000 shares at $13.50 per share on otherwise substantially the same terms as those set forth in the Securities Purchase Agreement.
At the close of the Private Placements on February 7, 2024, the Company received total gross proceeds of $127.8 million, before deducting placement agent fees and offering expenses.
Accumulated Other Comprehensive Loss
Accumulated other comprehensive loss included $0.5 million and $0.4 million of accumulated currency translation adjustments as of December 31, 2024 and 2023, respectively.
Equity Incentive Plans
In July 2014, the Company’s board of directors and its stockholders approved the establishment of the 2014 Equity Incentive Award Plan (the “2014 Plan”). The 2014 Plan provided for annual increases in the number of shares available for issuance thereunder on the first business day of each fiscal year, beginning with the year ended December 31, 2015, equal to four percent (4%) of the number of shares of the Company’s common stock outstanding as of such date or a lesser number of shares as determined by the Company’s board of directors. In June 2024, the Company adopted the 2024 Equity Incentive Award Plan (the “2024 Plan”). The Company reserved 4,688,345 shares for issuance pursuant to awards under the 2024 Plan. As of the effective date of the 2024 Plan, no further awards may be granted under the 2014 Plan.
In October 2017, the Company adopted the 2017 Inducement Plan (the “Inducement Plan”). The Company originally reserved 600,000 shares for issuance pursuant to stock options and RSUs under the Inducement Plan. The only persons eligible to receive grants of stock options and RSUs under the Inducement Plan are individuals who satisfy the standards for inducement grants under Nasdaq guidance, that is, generally, a person not previously an employee or director of Adverum, or following a bona fide period of non-employment, as an inducement material to the individual’s entering into employment with Adverum.
The 2014 Plan, 2024 Plan and Inducement Plan are referred to collectively as the Plans. As of December 31, 2024, a total of 1,277,999 shares were available for future issuance under the Plans.
Stock Options
Stock options under the 2024 Plan and the Inducement Plan may be granted for periods of up to 10 years and at prices no less than 100% of the estimated fair value of the shares on the date of grant as determined by the board of directors, provided, however, that the exercise price of an ISO and NSO granted to a 10% stockholder may not be less than 110% of the estimated fair value of the shares on the date of grant. Stock options granted to employees and non-employees generally vest ratably over four years.
The following table summarizes stock option activity under the Company’s stock plans and related information:
(In thousands, except exercise prices and years)Options
Outstanding
Weighted-
Average
Exercise
Price
Weighted-
Average
Remaining Contract
Life (in years)
Aggregate
Intrinsic Value (a)
Balance at December 31, 20232,303 $48.70 6.8$99 
Granted1,971 10.39 
Exercised(15)9.97 
Canceled/forfeited(350)50.28 
Balance at December 31, 20243,909 $29.39 8.1$— 
Vested and expected to vest as of December 31, 20243,909 $29.39 8.1$— 
Exercisable at December 31, 20241,504 $58.95 6.5$— 
(a)The aggregate intrinsic value is calculated as the difference between the stock option exercise price and the closing price of the Company’s common stock as quoted on a national exchange.
The total intrinsic value of stock options exercised during the years ended December 31, 2024 and 2023 was not material.
The fair value of each stock option issued was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions:
OptionsEmployee Stock Purchase Plan
Years ended December 31,Years ended December 31,
2024202320242023
Expected volatility95%90%83%78%
Expected term (in years)6.06.01.31.3
Expected dividend yield
Risk-free interest rate3.9%4.2%4.3%5.0%
The weighted-average fair values of options granted during the years ended December 31, 2024 and 2023 were $8.12 and $7.96, respectively.
As of December 31, 2024, there was $17.1 million of unrecognized stock-based compensation expense related to stock options that was expected to be recognized over a weighted-average period of 2.6 years.
RSUs
RSUs are share awards that entitle the holder to receive freely tradable shares of the Company’s common stock upon vesting. The fair value of RSUs is based upon the closing sales price of the Company’s common stock on the grant date. RSUs granted to employees generally vest over a three-year period.
The following table summarizes the RSU activity under the Company’s stock plans and related information:
(In thousands, except grant date fair value and years)Number of
Units
Weighted-
Average
Grant Date
Fair Value
Weighted-
Average
Remaining
Contractual Term
(in years)
Balance at December 31, 2023154 21.73 1.4
Granted160 12.04 
Vested and released(29)26.20 
Forfeited(26)14.22 
Balance at December 31, 2024259 $16.00 1.2
During the years ended December 31, 2024 and 2023, total fair value of RSUs vested was $0.8 million and $1.5 million, respectively. The number of RSUs vested includes shares of common stock that the Company withheld on behalf of employees or sold to cover to satisfy the minimum statutory tax withholding requirements. As of December 31, 2024, there was $1.7 million of unrecognized compensation cost related to unvested RSUs that is expected to be recognized over a weighted-average period of 1.8 years.
ESPP
In July 2014, the Company approved the establishment of the 2014 Employee Stock Purchase Plan (the “ESPP”). The Company reserved 208,833 shares of its common stock for issuance and provided for annual increases in the number of shares available for issuance on the first business day of each fiscal year, beginning in 2015, equal to the lesser of one percent (1%) of the number of the Company’s common stock shares outstanding as of such date or a number of shares as determined by the Company’s board of directors. During the years ended December 31, 2024 and 2023, 88,199 and 82,422 shares were issued under the ESPP, respectively. As of December 31, 2024, a total of 533,978 shares of common stock were available for future issuance under the ESPP. As of December 31, 2024, there was $0.5 million of unrecognized compensation cost related to the ESPP.
Stock-Based Compensation Recognized in the Consolidated Statements of Operations and Comprehensive Loss
The following table presents the Company’s stock-based compensation expense:
Years ended December 31,
20242023
(In thousands)
Research and development$4,544 $4,969 
General and administrative9,806 12,600 
Total share-based compensation expense$14,350 $17,569 
v3.25.1
401(k) Savings Plan
12 Months Ended
Dec. 31, 2024
Retirement Benefits [Abstract]  
401(k) Savings Plan 401(k) Savings Plan
The Company established a defined-contribution savings plan under Section 401(k) of the Code. The 401(k) Plan covers all employees who meet defined minimum age and service requirements, and allows participants to defer a portion of their annual compensation on a pretax basis. The amount of contributions that the Company made to the 401(k) Plan during the years ended December 31, 2024 and 2023 was $0.9 million.
v3.25.1
Segment Reporting
12 Months Ended
Dec. 31, 2024
Segment Reporting [Abstract]  
Segment Reporting Segment Reporting
Segment Reporting
The CODM assesses performance for the Company’s single reportable segment and decides how to allocate resources based on the Company’s total operating expenses as reported on the consolidated statements of operations and comprehensive loss. The CODM’s review of total operating expenses at the consolidated level is used to monitor the Company’s spending as well as budget versus actual results. The Company’s reportable segment primarily generates revenue through its license agreements (see Note 5).
As part of the CODM’s review of the segment’s performance, the CODM reviews the Company’s operating expense information, grouped by certain detailed line items from the internal income statement reporting. This includes research and development costs as well as general and administrative expenses. Based upon the operating expense information, the CODM can reconcile to net loss as reported on the consolidated statements of operations and comprehensive loss, shown in the table below.
The following table presents the segment loss, including significant segment expenses, for the years ended December 31, 2024 and 2023 (in thousands):
Years ended December 31,
20242023 As Restated
(In thousands)
License Revenue$1,000 $3,600 
Less:
Compensation and benefits expenses
56,546 54,572 
Clinical trial and manufacturing
22,333 20,235 
Facilities and IT
37,841 39,114 
Other research and development expense
4,728 4,985 
Other segment expenses(a)
18,711 13,636 
Total operating expenses140,159 132,542 
Operating loss(139,159)(128,942)
Other income, net
8,232 5,748 
Net loss before income taxes(130,927)(123,194)
Income tax benefit (provision)— 1,078 
Segment and consolidated net loss$(130,927)$(122,116)
(a)
Other segment expenses include professional services, consultants and contractors, travel and entertainment expenses, and general business expenses.
As of December 31, 2024 and 2023, all of the Company’s property and equipment was maintained in the United States. For each of the years ended December 31, 2024 and 2023, all of the Company’s license revenue was generated in the United States.
v3.25.1
Income Taxes
12 Months Ended
Dec. 31, 2024
Income Tax Disclosure [Abstract]  
Income Taxes Income Taxes
As part of the restatement, the Company adjusted the 2023 California research and development tax credit amounts as a result of a research and development credit study performed during 2024. This resulted in a net decrease of $3.4 million in the deferred tax asset balance related to the California research and development tax credit (net of a decrease in unrecognized tax benefits of $1.9 million) and a corresponding decrease in valuation allowance in 2023.
The following table presents domestic and foreign components of loss before provision for income taxes:
Years ended December 31,
20242023 As Restated
(In thousands)
U.S.$(130,768)$(123,040)
Foreign(159)(154)
Loss before income taxes$(130,927)$(123,194)
The components of the Company’s income tax (benefit) provision were as follows:
Years ended December 31,
20242023 As Restated
(In thousands)
Current:
Foreign$— $(1,078)
Total current tax (benefit) provision— (1,078)
Deferred
Foreign— — 
Total deferred tax provision— — 
Total income tax (benefit) provision$— $(1,078)
The income tax provision for the years ended December 31, 2024 and 2023 differed from the amounts computed by applying the statutory federal income tax rate of 21% to pretax loss as a result of the following:
Years ended December 31,
20242023 As Restated
(In thousands)
Federal income tax benefit at statutory rate$(27,495)$(25,871)
Stock compensation3,189 6,001 
Non-deductible expenses41 21 
Research and development tax credits(5,642)(1,810)
Change in valuation allowance23,801 19,558 
Foreign rate differential(14)(14)
Impact of internal reorganization— 1,323 
Uncertain tax positions6,100 (1,078)
Other20 792 
Total tax (benefit) provision$— $(1,078)
Deferred income taxes reflect the net tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The following table presents significant components of the Company’s deferred tax assets and liabilities:
As of December 31,
20242023 As Restated
(In thousands)
Deferred tax assets:
Net operating loss carryforwards$137,140 $128,171 
Accruals, reserve and other1,972 1,749 
Tax credit carryforwards29,263 21,254 
Stock-based compensation6,349 7,274 
Intangibles11 
Lease obligation19,622 17,322 
Capital losses9,850 9,850 
Section 174 R&D capitalization35,842 29,688 
Other
Total deferred tax assets before valuation allowance240,050 215,322 
Valuation allowance(232,058)(205,861)
Total deferred tax assets7,992 9,461 
Deferred tax liabilities:
Right-of-use assets(7,149)(8,153)
Property and equipment(843)(1,308)
Total deferred tax liabilities$(7,992)$(9,461)
Net deferred tax assets$— $— 
The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. Based on the Company’s history of operating losses, the Company has concluded that it is more likely than not that the benefit of its deferred tax assets will not be realized. Accordingly, the Company has provided a full valuation allowance for deferred tax assets as of December 31, 2024 and 2023. The valuation allowance increased approximately $26.2 million and $17.7 million during the years ended December 31, 2024 and 2023, respectively, mainly driven by net operating losses (“NOLs”).
As of December 31, 2024, the Company had U.S. federal NOLs carryforwards of approximately $552.7 million to offset any future federal income. Approximately $57.3 million of NOLs expire at various years beginning with 2032. As of December 31, 2024, the Company also had U.S. state NOL carryforwards of approximately $345.2 million to offset any future state income. U.S. state NOLs expire at various years beginning with 2037. At December 31, 2024, the Company also had approximately $49.5 million of foreign net operating loss carryforwards which may be available to offset future foreign income; these carryforwards do not expire.
As of December 31, 2024, the Company had federal research and development tax credit carryforwards of approximately $25.1 million available to reduce future tax liabilities which expire at various years beginning with 2036. As of December 31, 2024, the Company had state credit carryforwards of approximately $13.8 million available to reduce future tax liabilities which do not expire.
Under Section 382 and 383 of the Code, our ability to utilize NOL carryforwards or other tax attributes such as research tax credits, in any taxable year may be limited if the Company has experienced an “ownership change.” Generally, a Section 382 ownership change occurs if there is a cumulative increase of more than 50 percentage points in the stock ownership of one or more stockholders or groups of stockholders who own at least 5% of a corporation’s stock within a specified testing period. Similar rules may apply under state tax laws. The Company is conducting a Section 382 study, which is still subject to finalization, but which may show that the Company experienced an ownership change in 2024. If such an ownership change has occurred then up to vast majority of the NOLs, capital losses and research credit carryforwards presented in the consolidated financial statements would be subject to limitations and therefore may expire unutilized.
The Company files income tax returns in the U.S. federal, state, and foreign jurisdictions. The federal, state and foreign income tax returns are open under the statute of limitations subject to tax examinations for the tax years ended December 31, 2019 through December 31, 2023. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, state or foreign tax authorities to the extent utilized in a future period.
The Company has total unrecognized tax benefits as of December 31, 2024 and 2023 of approximately $27.2 million and $22.8 million, respectively. If the unrecognized tax benefits for uncertain tax positions as of December 31, 2023, is recognized, there will be no impact to the effective tax rate as the tax benefit would increase the net deferred tax assets, which is currently offset with a full valuation allowance. A reconciliation of the unrecognized tax benefits is as follows:
Years ended December 31,
20242023 As Restated
(In thousands)
Unrecognized tax benefits as of the beginning of the year$22,758 $23,069 
Increase related to tax positions taken during the prior year— 43 
Decrease related to tax positions taken during the prior year
(2,880)— 
Increase related to tax position taken during the current year
7,310 3,121 
Decrease related to tax position taken during the current year— (3,475)
Unrecognized tax benefits as of the end of the year$27,188 $22,758 
The Company recognizes interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2024 and 2023, the Company had no accrued interest and penalties related to uncertain tax positions and no amount has been recognized in the Company's consolidated statement of operations and comprehensive loss. There are no ongoing examinations by taxing authorities at this time.
v3.25.1
Net Loss per Share
12 Months Ended
Dec. 31, 2024
Earnings Per Share [Abstract]  
Net Loss per Share Net Loss per Share
For the year ended December 31, 2024, the Pre-Funded Warrants to purchase the Company's shares of common stock issued in the Private Placements were included in the basic and diluted net loss per share calculation as the exercise price is non-substantive and virtually assured.
The following common stock equivalents outstanding at the end of the periods presented were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect:
As of December 31,
20242023
(In thousands)
Stock options3,909 2,303 
Restricted stock units259 154 
ESPP32 17 
4,200 2,474 
v3.25.1
Quarterly Financial Information (Unaudited)
12 Months Ended
Dec. 31, 2024
Quarterly Financial Information Disclosure [Abstract]  
Quarterly Financial Information Quarterly Financial Information (Unaudited)
The previously reported financial information for the three months ended March 31, 2024, three and six months ended June 30, 2024 and the three and nine months ended September 30, 2024 and the comparable periods in 2023, has been restated. Relevant restated financial information for each period is included in this restatement and for other immaterial adjustments in the tables that follow.
Restatement Related to NC Premises
As described in Note 2, the Company inappropriately omitted accounting for available tenant improvement allowance that resulted in a misstatement of its operating lease right-of-use asset and related lease liability. Additionally, the Company did not account for a tenant improvement allowances that were conveyed to the subtenant resulting in an overstatement of sublease income. The tables below reflect the adjustments related to this restatement, the amounts of which are included with reference (a).
During the quarter ended June 30, 2024, the Company inappropriately assessed the collectibility of the sublease payments as probable and recorded a deferred rent receivable of $7.0 million. The Company corrected its collectibility assessment and recognized an adjustment to eliminate the $7.0 million and $7.1 million deferred rent receivable in the second and third quarters of 2024, respectively, the amounts of which are included in the tables below with reference (b).
Other Adjustments
As part of this restatement, the Company has corrected certain other immaterial errors. These adjustments are described in more detail references (c) and (d) as follows:
(c)The Company had previously recorded an out of period adjustment related to research and development expenses in the first quarter of 2023 that was the result of not recording a severance accrual of $0.2 million in 2022.
(d)The Company had previously understated its right-of-use assets and lease liability by $0.3 million related to its facilities in Redwood City, California at each balance sheet date in 2024 and 2023.
The unaudited interim financial statements reflect all adjustments which are, in the opinion of management, necessary for a fair statement of the results for the interim periods presented (in thousands, except per share data).
Condensed Consolidated Balance Sheets
September 30, 2024 (unaudited)
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$92,851 $— $92,851 
Short-term investments
60,390 — 60,390 
Prepaid expenses and other current assets7,839 — 7,839 
Total current assets161,080 — 161,080 
Operating lease right-of-use assets50,695 (17,015)
(a) (d)
33,680 
Property and equipment, net
12,215 — 12,215 
Deferred rent receivable
7,116 (7,116)
(b)
— 
Restricted cash
1,976 — 1,976 
Deposit and other long-term assets
1,293 — 1,293 
Total assets$234,375 $(24,131)$210,244 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
$2,110 — $2,110 
Accrued expenses and other current liabilities14,886 — 14,886 
Lease liability, current portion
10,661 (5,044)
(a)
5,617 
Total current liabilities27,657 (5,044)22,613 
Long-term liabilities:
Lease liability, net of current portion62,602 16,759 
(a) (d)
79,361 
Total liabilities90,259 11,715 101,974 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital1,134,556 — 1,134,556 
Accumulated other comprehensive loss(268)— (268)
Accumulated deficit(990,174)(35,846)
(a) (b)
(1,026,020)
Total stockholders’ equity144,116 (35,846)108,270 
Total liabilities and stockholders’ equity$234,375 $(24,131)$210,244 
June 30, 2024 (unaudited)
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$127,321 $— $127,321 
Short-term investments
46,506 — 46,506 
Prepaid expenses and other current assets5,591 — 5,591 
Total current assets179,418 — 179,418 
Operating lease right-of-use assets51,237 (17,241)
(a) (d)
33,996 
Property and equipment, net
13,047 13,047 
Deferred rent receivable
6,963 (6,963)
(b)
— 
Restricted cash
1,976 — 1,976 
Deposit and other long-term assets
1,162 — 1,162 
Total assets$253,803 $(24,204)$229,599 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
1,973 — 1,973 
Accrued expenses and other current liabilities9,912 — 9,912 
Lease liability, current portion
10,576 (5,006)
(a)
5,570 
Total current liabilities22,461 (5,006)17,455 
Long-term liabilities:
Lease liability, net of current portion63,313 11,431 
(a) (d)
74,744 
Total liabilities85,774 6,425 92,199 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital1,131,592 — 1,131,592 
Accumulated other comprehensive loss(525)— (525)
Accumulated deficit(963,040)(30,629)
(a) (b)
(993,669)
Total stockholders’ equity168,029 (30,629)137,400 
Total liabilities and stockholders’ equity$253,803 $(24,204)$229,599 
March 31, 2024 (unaudited)
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$149,608 $— $149,608 
Short-term investments
43,720 — 43,720 
Prepaid expenses and other current assets5,802 — 5,802 
Total current assets199,130 — 199,130 
Operating lease right-of-use assets51,760 (17,282)
(a) (d)
34,478 
Property and equipment, net
13,766 — 13,766 
Restricted cash
1,976 — 1,976 
Deposit and other long-term assets
1,196 — 1,196 
Total assets$267,828 $(17,282)$250,546 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
1,897 — 1,897 
Accrued expenses and other current liabilities9,160 — 9,160 
Lease liability, current portion
10,492 (4,971)
(a)
5,521 
Total current liabilities21,549 (4,971)16,578 
Long-term liabilities:
Lease liability, net of current portion63,991 6,296 
(a) (d)
70,287 
Total liabilities85,540 1,325 86,865 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital1,127,383 — 1,127,383 
Accumulated other comprehensive loss(533)— (533)
Accumulated deficit(944,564)(18,607)
(a)
(963,171)
Total stockholders’ equity182,288 (18,607)163,681 
Total liabilities and stockholders’ equity$267,828 $(17,282)$250,546 
September 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$105,366 $— $105,366 
Short-term investments
11,716 — 11,716 
Prepaid expenses and other current assets7,514 — 7,514 
Total current assets124,596 — 124,596 
Operating lease right-of-use assets52,757 (17,158)
(a) (d)
35,599 
Property and equipment, net
15,497 — 15,497 
Restricted cash
2,650 — 2,650 
Deposit and other long-term assets
1,270 — 1,270 
Total assets$196,770 $(17,158)$179,612 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
2,170 — 2,170 
Accrued expenses and other current liabilities16,362 — 16,362 
Lease liability, current portion
10,324 (4,897)
(a)
5,427 
Total current liabilities28,856 (4,897)23,959 
Long-term liabilities:
Lease liability, net of current portion65,200 3,196 
(a) (d)
68,396 
Total liabilities94,056 (1,701)92,355 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital999,328 — 999,328 
Accumulated other comprehensive loss(552)— (552)
Accumulated deficit(896,063)(15,457)
(a) (c)
(911,520)
Total stockholders’ equity102,714 (15,457)87,257 
Total liabilities and stockholders’ equity$196,770 $(17,158)$179,612 
June 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$111,187 $— $111,187 
Short-term investments
30,266 — 30,266 
Prepaid expenses and other current assets5,690 — 5,690 
Total current assets147,143 — 147,143 
Operating lease right-of-use assets58,287 (17,054)
(a) (d)
41,233 
Property and equipment, net
32,041 — 32,041 
Restricted cash
2,650 — 2,650 
Deposit and other long-term assets
1,308 — 1,308 
Total assets$241,429 $(17,054)$224,375 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
1,418 — 1,418 
Accrued expenses and other current liabilities17,704 — 17,704 
Lease liability, current portion
25,396 (4,861)
(a)
20,535 
Total current liabilities44,518 (4,861)39,657 
Long-term liabilities:
Lease liability, net of current portion65,756 2,734 
(a) (d)
68,490 
Total liabilities110,274 (2,127)108,147 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital994,938 — 994,938 
Accumulated other comprehensive loss(606)— (606)
Accumulated deficit(863,178)(14,927)
(a) (c)
(878,105)
Total stockholders’ equity131,155 (14,927)116,228 
Total liabilities and stockholders’ equity$241,429 $(17,054)$224,375 
March 31, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$67,552 $— $67,552 
Short-term investments
96,733 — 96,733 
Prepaid expenses and other current assets4,588 — 4,588 
Total current assets168,873 — 168,873 
Operating lease right-of-use assets69,503 (22,905)
(a) (d)
46,598 
Property and equipment, net
33,347 — 33,347 
Restricted cash
2,503 — 2,503 
Deposit and other long-term assets
1,351 — 1,351 
Total assets$275,577 $(22,905)$252,672 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
2,863 — 2,863 
Accrued expenses and other current liabilities17,344 — 17,344 
Lease liability, current portion
26,265 (5,531)
(a)
20,734 
Total current liabilities46,472 (5,531)40,941 
Long-term liabilities:
Lease liability, net of current portion71,348 (3,462)
(a) (d)
67,886 
Total liabilities117,820 (8,993)108,827 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital990,223 — 990,223 
Accumulated other comprehensive loss(799)— (799)
Accumulated deficit(831,668)(13,912)
(a) (c)
(845,580)
Total stockholders’ equity157,757 (13,912)143,845 
Total liabilities and stockholders’ equity$275,577 (22,905)$252,672 
Quarterly and Year to Date Condensed Consolidated Statements of Operations and Comprehensive Loss
Three Months Ended
Nine Months Ended
September 30, 2024 (unaudited)September 30, 2024 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
License revenue$1,000 $— $1,000 $1,000 $— $1,000 
Operating expenses:
Research and development20,439 — 20,439 52,946 — 52,946 
General and administrative9,782 5,217 
(a)
14,999 24,996 19,599 
(a) (b)
44,595 
Total operating expenses30,221 5,217 35,438 77,942 19,599 97,541 
Operating loss(29,221)(5,217)(34,438)(76,942)(19,599)(96,541)
Other income, net2,087— 2,0876,545— 6,545
Net loss(27,134)(5,217)(32,351)(70,397)(19,599)(89,996)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities241 — 241 198 — 198 
Foreign currency translation adjustment16 — 16 — 
Comprehensive loss$(26,877)$(5,217)$(32,094)$(70,192)$(19,599)$(89,791)
Net loss per share — basic and diluted$(1.30)— $(1.55)$(3.63)— $(4.64)
Weighted-average common shares used to compute net loss per share - basic and diluted20,876 — 20,876 19,408 — 19,408 

Three Months Ended
Six Months Ended
June 30, 2024 (unaudited)June 30, 2024 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
Operating expenses:
Research and development$17,097 $— $17,097 $32,507 $— $32,507 
General and administrative3,785 12,022 
(a) (b)
15,807 15,214 14,382 
(a) (b)
29,596 
Total operating expenses20,882 12,022 32,904 47,721 14,382 62,103 
Operating loss(20,882)(12,022)(32,904)(47,721)(14,382)(62,103)
Other income, net2,406 — 2,406 4,458 — 4,458 
Net loss(18,476)(12,022)(30,498)(43,263)(14,382)(57,645)
Other comprehensive gain (loss):
Net unrealized loss on marketable securities
(2)— (2)(43)— (43)
Foreign currency translation adjustment10 — 10 (9)— (9)
Comprehensive loss$(18,468)(12,022)$(30,490)$(43,315)$(14,382)$(57,697)
Net loss per share — basic and diluted$(0.89)— $(1.46)$(2.31)— $(3.08)
Weighted-average common shares used to compute net loss per share - basic and diluted20,852 — 20,852 18,713  18,713 
Three Months Ended
March 31, 2024 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Operating expenses:
Research and development$15,410 $— $15,410 
General and administrative11,429 2,360 
(a)
13,789 
Total operating expenses26,839 2,360 29,199 
Operating loss(26,839)(2,360)(29,199)
Other income, net2,052 — 2,052 
Net loss(24,787)(2,360)(27,147)
Other comprehensive gain (loss):
Net unrealized loss on marketable securities
(41)— (41)
Foreign currency translation adjustment(19)— (19)
Comprehensive loss$(24,847)$(2,360)$(27,207)
Net loss per share — basic and diluted$(1.50)— $(1.65)
Weighted-average common shares used to compute net loss per share - basic and diluted16,479 — 16,479 

Three Months EndedNine Months Ended
September 30, 2023 (unaudited)September 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
License revenue$— $— $— $3,600 $— $3,600 
Operating expenses:
Research and development20,740 — 20,740 62,398 (190)
(c)
62,208 
General and administrative13,789 530 
(a)
14,319 39,035 4,351 
(a)
43,386 
Total operating expenses34,529 530 35,059 101,433 4,161 105,594 
Operating loss(34,529)(530)(35,059)(97,833)(4,161)(101,994)
Other income, net1,661— 1,6614,437— 4,437
Net loss before income taxes(32,868)(530)(33,398)(93,396)(4,161)(97,557)
Income tax provision(17)(17)(55)(55)
Net loss(32,885)(530)(33,415)(93,451)(4,161)(97,612)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities66 — 66 1,003 — 1,003 
Foreign currency translation adjustment(12)— (12)(24)— (24)
Comprehensive loss$(32,831)(530)$(33,361)$(92,472)$(4,161)$(96,633)
Net loss per share — basic and diluted$(3.26)— $(3.31)$(9.28)— $(9.69)
Weighted-average common shares used to compute net loss per share - basic and diluted10,099 — 10,099 10,069 — 10,069 
Three Months Ended
Six Months Ended
June 30, 2023 (unaudited)June 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
License revenue$— $— $— $3,600 $— $3,600 
Operating expenses:
Research and development$20,599 $— $20,599 $41,658 $(190)
(c)
$41,468 
General and administrative12,466 1,015 
(a)
13,481 25,246 3,821 
(a)
29,067 
Total operating expenses33,065 1,015 34,080 66,904 3,631 70,535 
Operating loss(33,065)(1,015)(34,080)(63,304)(3,631)(66,935)
Other income, net1,576 — 1,576 2,776 — 2,776 
Net loss before income taxes(31,489)(1,015)(32,504)(60,528)(3,631)(64,159)
Income tax provision(21)(21)(38)(38)
Net loss(31,510)(1,015)(32,525)(60,566)(3,631)(64,197)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities
198 — 198 937 — 937 
Foreign currency translation adjustment(5)— (5)(12)— (12)
Comprehensive loss$(31,317)(1,015)$(32,332)$(59,641)$(3,631)$(63,272)
Net loss per share — basic and diluted$(3.13)— $(3.23)$(6.02)— $(6.39)
Weighted-average common shares used to compute net loss per share - basic and diluted10,076 — 10,076 10,053 — 10,053 

Three Months Ended
March 31, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
License revenue$3,600 $— $3,600 
Operating expenses:
Research and development$21,059 $(190)
(c)
$20,869 
General and administrative12,780 2,806 
(a)
15,586 
Total operating expenses33,839 2,616 36,455 
Operating loss(30,239)(2,616)(32,855)
Other income, net1,200 — 1,200 
Net loss before income taxes(29,039)(2,616)(31,655)
Income tax provision(17)— (17)
Net loss(29,056)(2,616)(31,672)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities
739 — 739 
Foreign currency translation adjustment(7)— (7)
Comprehensive loss$(28,324)(2,616)$(30,940)
Net loss per share — basic and diluted$(2.90)— $(3.16)
Weighted-average common shares used to compute net loss per share - basic and diluted
10,030 — 10,030 
Statements of Cash Flows
The effects of this restatement on certain line items of the condensed consolidated statements of cash flows are summarized in the following tables (in thousands). There was no impact on net cash used in operating activities or within any line items within investing and financing activities.
Nine Months Ended September 30, 2024
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(70,397)$(19,599)
(a) (b)
$(89,996)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense1,571 4,006 
(a)
5,577 
Non-cash sublease loss
— 8,477 
(a)
8,477 
Changes in operating assets and liabilities:
Deposit and other long-term assets and deferred rent receivable(7,178)7,116 
(b)
(62)

Six Months Ended June 30, 2024
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(43,263)$(14,382)
(a) (b)
$(57,645)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense1,029 2,654 
(a)
3,683 
Non-cash sublease loss
— 4,765 
(a)
4,765 
Changes in operating assets and liabilities:
Deposit and other long-term assets and deferred rent receivable(6,894)6,963 
(b)
69 

Three Months Ended March 31, 2024
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(24,787)$(2,360)
(a)
$(27,147)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense506 1,314 
(a)
1,820 
Non-cash sublease loss
— 1,046 
(a)
1,046 
Nine Months Ended September 30, 2023
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(93,451)$(4,161)
(a) (c)
$(97,612)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense12,466 3,916 
(a)
16,382 
Non-cash sublease loss
— 435 
(a)
435 
Changes in operating assets and liabilities:— 
Accrued expenses and other liabilities(1,452)(190)
(c)
(1,642)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets
$13,711 $(6,079)$7,632 

Six Months Ended June 30, 2023
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(60,566)$(3,631)
(a) (c)
(64,197)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense6,936 2,602 
(a)
9,538 
Non-cash sublease loss
— 1,219 
(a)
1,219 
Changes in operating assets and liabilities:
Accrued expenses and other liabilities(525)(190)
(c)
(715)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets
$13,711 $(6,079)$7,632 
Three Months Ended March 31, 2023
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(29,056)$(2,616)
(a) (c)
$(31,672)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense1,427 1,239 
(a)
2,666 
Non-cash sublease loss
— 1,567 1,567 
Changes in operating assets and liabilities:— 
Accrued expenses and other liabilities(749)(190)
(c)
(939)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets
$8,004 $(104)$7,900 
v3.25.1
Subsequent Events
12 Months Ended
Dec. 31, 2024
Subsequent Events [Abstract]  
Subsequent Events Subsequent Events
In February 2025, a lien in the amount of $4.8 million was filed by a third-party contractor against the subtenant's interest in the NC Premises. The Company discharged the lien in March 2025 by depositing cash in the full amount with the applicable court in order to avoid a default under the head lease. Subsequently, the subtenant and Jaguar failed to remit the March 2025 rent and subsequent rent payments and defaulted, and failed to cure such defaults, under the sublease and the assignment agreement for the NC Premises.
As a result, the Company assumed responsibility for such payments in March 2025 and as of April 7, 2025 had made a total amount of $1.9 million to satisfy outstanding rent and common area management fee obligations. The Company remains obligated under the head lease, including for all future remaining rent payments in an aggregate amount of up to $119.7 million for the remainder of the head lease term, of which $5.7 million is due between April 8 and December 31, 2025. As a result of the uncured defaults, the Company terminated the sublease and initiated a lawsuit against the subtenant and Jaguar in the Superior Court of Wake County, North Carolina to enforce its rights under the sublease and to seek recovery of losses and damages incurred due to the defaults by the subtenant and Jaguar.
v3.25.1
Pay vs Performance Disclosure - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Pay vs Performance Disclosure                        
Net loss $ (32,351) $ (30,498) $ (27,147) $ (33,415) $ (32,525) $ (31,672) $ (57,645) $ (64,197) $ (89,996) $ (97,612) $ (130,927) $ (122,116)
v3.25.1
Insider Trading Arrangements
3 Months Ended
Dec. 31, 2024
Trading Arrangements, by Individual  
Rule 10b5-1 Arrangement Adopted false
Non-Rule 10b5-1 Arrangement Adopted false
Rule 10b5-1 Arrangement Terminated false
Non-Rule 10b5-1 Arrangement Terminated false
v3.25.1
Insider Trading Policies and Procedures
12 Months Ended
Dec. 31, 2024
Insider Trading Policies and Procedures [Line Items]  
Insider Trading Policies and Procedures Adopted true
v3.25.1
Cybersecurity Risk Management and Strategy Disclosure
12 Months Ended
Dec. 31, 2024
Cybersecurity Risk Management, Strategy, and Governance [Line Items]  
Cybersecurity Risk Management Processes for Assessing, Identifying, and Managing Threats [Text Block]
Risk Management and Strategy
We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third-party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, confidential information that is proprietary, strategic or competitive in nature, and clinical trial data (“Information Systems and Data”).
Our information security function, which includes our Vice President of Information Technology (“IT”) and third-party service providers, legal department and security committee, helps identify, assess, and manage the Company’s cybersecurity threats and risks. Our information security function identifies and assesses risks from cybersecurity threats by monitoring and evaluating our threat environment using various methods including, for example: using automated tools, conducting audits, evaluating threats, and performing vulnerability assessments.
Depending on the environment, systems, and data at issue, we implement and maintain various technical, physical, and organizational measures, processes, and policies designed to manage and mitigate material risks from cybersecurity threats to our Information Systems and Data, including, for example: cybersecurity insurance, physical security, incident detection and response, access controls, employee training, penetration testing, and systems monitoring.
Our assessment and management of material risks from cybersecurity threats are integrated into the Company’s overall risk management processes. For example, our Vice President of IT, works with management to prioritize our risk management processes and mitigate cybersecurity threats that are more likely to lead to a material impact to our business.
We use third-party service providers to assist us from time to time to identify, assess, and manage material risks from cybersecurity threats, including for example: cybersecurity software providers, managed cybersecurity service providers, penetration testing firms, and cybersecurity consultants.
We use third-party service providers to perform a variety of functions throughout our business, such as contract research organizations, contract manufacturing organizations, and supply chain resources. We have a vendor management process to manage cybersecurity risks associated with certain of these providers. Depending on the nature of the services provided, the sensitivity of the Information Systems and Data at issue, and the identity of the provider, our vendor management process may involve different levels of diligence designed to help identify cybersecurity risks associated with a provider such as risk assessments and reviewing security assessments and reports.
For a description of the risks from cybersecurity threats that may materially affect the Company and how they may do so, see our risk factors under Part 1. Item 1A. Risk Factors in this Annual Report on Form 10-K, including the risk factor entitled: If our information technology systems or those third parties with whom we work, or our data, are or were compromised, we could experience adverse consequences resulting from such compromise, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; material disruption of our product development programs; and other adverse consequences.
Cybersecurity Risk Management Processes Integrated [Flag] true
Cybersecurity Risk Management Processes Integrated [Text Block]
We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third-party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, confidential information that is proprietary, strategic or competitive in nature, and clinical trial data (“Information Systems and Data”).
Cybersecurity Risk Management Third Party Engaged [Flag] true
Cybersecurity Risk Third Party Oversight and Identification Processes [Flag] true
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Flag] false
Cybersecurity Risk Board of Directors Oversight [Text Block]
Governance
Our Board of Directors addresses the Company’s cybersecurity risk management as part of its general oversight function. The Board of Directors is responsible for overseeing Company’s cybersecurity risk management processes, including oversight of mitigation of risks from cybersecurity threats.
Our cybersecurity risk assessment and management processes are implemented and maintained by certain Company management, including our Vice President of IT, who holds a Bachelor of Science in Computer Methods from California State University-Long Beach and has previously served as a Director and Associate Director of IT for large pharmaceutical companies.
Our management, including our Chief Operating Officer and Vice President of IT, is responsible for hiring appropriate information security personnel, helping to integrate cybersecurity risk considerations into the Company’s overall risk management strategy, and helping prepare for cybersecurity incidents.
Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Vice President of IT and General Counsel, who work with the Company’s incident response team to help the Company mitigate and remediate cybersecurity incidents of which they are notified. In addition, the Company’s incident response process includes reporting to the Audit Committee of the Board of Directors for certain cybersecurity incidents.
The Board of Directors receives periodic reports from the management concerning the Company’s significant cybersecurity threats and risk and the processes the Company has implemented to address them. The Board of Directors also has access to various reports, summaries or presentations related to cybersecurity threats, risk, and mitigation.
Cybersecurity Risk Board Committee or Subcommittee Responsible for Oversight [Text Block]
Our Board of Directors addresses the Company’s cybersecurity risk management as part of its general oversight function. The Board of Directors is responsible for overseeing Company’s cybersecurity risk management processes, including oversight of mitigation of risks from cybersecurity threats.
Cybersecurity Risk Process for Informing Board Committee or Subcommittee Responsible for Oversight [Text Block]
Our Board of Directors addresses the Company’s cybersecurity risk management as part of its general oversight function. The Board of Directors is responsible for overseeing Company’s cybersecurity risk management processes, including oversight of mitigation of risks from cybersecurity threats.
Our cybersecurity risk assessment and management processes are implemented and maintained by certain Company management, including our Vice President of IT, who holds a Bachelor of Science in Computer Methods from California State University-Long Beach and has previously served as a Director and Associate Director of IT for large pharmaceutical companies.
Cybersecurity Risk Role of Management [Text Block]
Our management, including our Chief Operating Officer and Vice President of IT, is responsible for hiring appropriate information security personnel, helping to integrate cybersecurity risk considerations into the Company’s overall risk management strategy, and helping prepare for cybersecurity incidents.
Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Vice President of IT and General Counsel, who work with the Company’s incident response team to help the Company mitigate and remediate cybersecurity incidents of which they are notified. In addition, the Company’s incident response process includes reporting to the Audit Committee of the Board of Directors for certain cybersecurity incidents.
The Board of Directors receives periodic reports from the management concerning the Company’s significant cybersecurity threats and risk and the processes the Company has implemented to address them. The Board of Directors also has access to various reports, summaries or presentations related to cybersecurity threats, risk, and mitigation.
Cybersecurity Risk Management Positions or Committees Responsible [Flag] true
Cybersecurity Risk Management Positions or Committees Responsible [Text Block]
Our cybersecurity risk assessment and management processes are implemented and maintained by certain Company management, including our Vice President of IT, who holds a Bachelor of Science in Computer Methods from California State University-Long Beach and has previously served as a Director and Associate Director of IT for large pharmaceutical companies.
Our management, including our Chief Operating Officer and Vice President of IT, is responsible for hiring appropriate information security personnel, helping to integrate cybersecurity risk considerations into the Company’s overall risk management strategy, and helping prepare for cybersecurity incidents.
Cybersecurity Risk Management Expertise of Management Responsible [Text Block] Our cybersecurity risk assessment and management processes are implemented and maintained by certain Company management, including our Vice President of IT, who holds a Bachelor of Science in Computer Methods from California State University-Long Beach and has previously served as a Director and Associate Director of IT for large pharmaceutical companies
Cybersecurity Risk Process for Informing Management or Committees Responsible [Text Block] Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Vice President of IT and General Counsel, who work with the Company’s incident response team to help the Company mitigate and remediate cybersecurity incidents of which they are notified. In addition, the Company’s incident response process includes reporting to the Audit Committee of the Board of Directors for certain cybersecurity incidents.
Cybersecurity Risk Management Positions or Committees Responsible Report to Board [Flag] true
v3.25.1
Summary of Significant Accounting Policies (Policies)
12 Months Ended
Dec. 31, 2024
Accounting Policies [Abstract]  
Basis of Presentation and Principles of Consolidation
Basis of Presentation and Principles of Consolidation—The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). The consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions and balances have been eliminated in consolidation.
Use of Estimates
Use of Estimates—The preparation of financial statements in conformity with U.S. GAAP requires management to make judgements, estimates and assumptions that affect the reported amounts of assets, liabilities, revenue and expenses, and related disclosures. Management bases its estimates on historical experience and on various other assumptions that are believed to be reasonable under the circumstances. These estimates form the basis for making judgments about the carrying values of assets and liabilities when these values are not readily apparent from other sources. Accounting estimates and judgements are inherently uncertain, and the actual results could differ from these estimates.
March 2024 Reverse Stock Split
March 2024 Reverse Stock Split—On March 21, 2024, the Company effected a 1-for-10 reverse stock split of its common stock. The par value and the authorized shares of the common stock were not adjusted as a result of the reverse stock split.
All equity related information including per share amounts for all periods presented in these consolidated financial statements and the notes thereto have been adjusted retroactively, where applicable, to reflect this reverse stock split.
Foreign Currency
Foreign Currency—Assets and liabilities of non-U.S. subsidiaries that operate in a local currency environment, where the local currency is the functional currency, are translated to U.S. dollars at exchange rates in effect at the balance sheet date, with the resulting translation adjustments directly recorded to a separate component of accumulated other comprehensive loss. Upon sale or upon complete or substantially complete liquidation of an investment in a foreign entity, the amount attributable to that entity and accumulated in the translation adjustment component of equity is removed from the separate component of equity and reported as part of the gain or loss on sale or liquidation of the investment for the period during which the sale or liquidation occurs. Income and expense accounts are translated at average exchange rates for the period. Transactions which are not in the functional currency of the entity are remeasured into the functional currency and gains or losses resulting from the remeasurement recorded in other income, net.
Cash and Cash Equivalents
Cash and Cash Equivalents—The Company considers all highly liquid investments purchased with original maturities of three months or less at the date of purchase to be cash equivalents. Cash and cash equivalents include cash held in banks, money market accounts and highly liquid debt securities. Cash equivalents are stated at fair value.
Restricted Cash
Restricted Cash—Restricted cash primarily consists of cash collateral to letter of credit provided to the landlord in relation to a lease agreement. See Note 6, Leases for additional information.
Short-Term Investments
Short-Term Investments—All short-term investments in debt securities have been classified as “available for sale” and are carried at fair value. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive loss and reported as a separate component of stockholders’ equity until realized. The cost of securities sold is based on the specific-identification method. The amortized cost of securities is adjusted for amortization of premiums and accretion of discounts to maturity. Interest on short-term investments is included in other income, net in the Company’s consolidated statements of operations and comprehensive loss. In accordance with the Company’s investment policy, management invests to diversify credit risk and only invests in securities with high credit quality, including U.S. government securities.
The Company assesses available-for-sale debt securities on a quarterly basis to see whether any unrealized loss is due to credit-related factors. Factors considered in determining whether an impairment is credit-related include the extent to which the investment’s fair value is less than its cost basis, declines in published credit ratings, changes in interest rates, and any other adverse factors related to the security. If it is determined that a credit-related impairment exists, the Company will measure the credit loss based on a discounted cash flow model. Credit-related impairments on available-for-sale debt securities are recognized as an allowance for credit losses with a corresponding adjustment to other income, net in the Company’s consolidated statement of operations. The unrealized loss position that is not credit-related is recorded, net of any related tax effects, in other comprehensive income until realized. There were no credit-related losses recognized for the periods presented.
Accrued Interest Receivable
Accrued Interest Receivable—Accrued interest receivable related to the Company’s available-for-sale debt securities is presented within prepaid expenses and other current assets on the Company’s consolidated balance sheets. The Company has elected to exclude accrued interest receivable from both the fair value and the amortized cost basis of available-for-sale debt securities for the purposes of identifying and measuring any impairment. The Company writes off accrued interest receivable once it has determined that the asset is not realizable. Any write offs of accrued interest receivable are recorded by reversing interest income, recognizing credit loss expense, or a combination of both. To date, the Company has not written off any accrued interest receivables associated with its available-for-sale debt securities.
Segment Reporting
Segment Reporting—The Company operates and manages its business as one reporting and operating segment, which is the business of developing and commercializing gene therapeutics. The Company’s chief executive officer, who is the chief operating decision maker (“CODM”), assesses segment performance and decides how to allocate resources based on total operating expenses as reported on the consolidated statements of operations and comprehensive loss. See Note 11, Segment Reporting, for additional information.
Concentrations of Credit Risk and Other Uncertainties
Concentrations of Credit Risk and Other Uncertainties—Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash, cash equivalents and short-term investments. Risks associated with cash, cash equivalents, short-term investments are mitigated by the Company’s investment policy, which limits the Company’s investing to securities having specified credit ratings. Management believes that the Company is not exposed to significant credit risk.
The Company is subject to certain risks and uncertainties, including, but not limited to changes in any of the following areas that the Company believes could have a material adverse effect on future financial position or results of operations: the ability to obtain future financing; regulatory approval and market acceptance of, and reimbursement for, the Company’s product candidates; performance of third-party clinical research organizations and manufacturers; development of sales channels; protection of intellectual property; litigation or claims against the Company based on intellectual property, patent, product, regulatory or other factors; and the Company’s ability to attract and retain employees necessary to support growth.
Property and Equipment
Property and Equipment—Property and equipment are recorded at cost, net of accumulated depreciation and amortization. Depreciation is recorded using the straight-line method over the estimated useful lives of the assets, generally three to five years. Leasehold improvements are capitalized and amortized over the shorter period of their expected lives or the lease term. Major replacements and improvements are capitalized, while general repairs and maintenance are expensed as incurred.
Impairment of Long-Lived Assets
Impairment of Long-Lived Assets—Long-lived assets, including property and equipment and finite-lived intangible assets, are reviewed for impairment whenever facts or circumstances either internally or externally may indicate that the carrying value of an asset may not be recoverable. If there is an indication of impairment, the Company tests for recoverability by comparing the estimated undiscounted future cash flows expected to result from the use of the asset to the carrying amount of the asset or asset group. If the asset or asset group is determined to be impaired, any excess of the carrying value of the asset or asset group over its estimated fair value is recognized as an impairment loss.
Leases
Leases For long-term operating leases, the Company recognizes a right-of-use asset and a lease liability on the Company's consolidated balance sheets. The Company determines if an arrangement contains a lease and the classification of the lease at inception. An arrangement contains a lease if there is an identified asset and if the Company controls the use of the identified asset throughout the period of use. The lease liability is determined as the present value of future lease payments and lease incentives over the expected lease term. Lease incentives are included in the measurement of the consideration in the contract at lease commencement when the Company is reasonably certain to incur costs to construct lessee assets. In order to determine the incremental borrowing rate, the Company determines its credit rating, adjusts the credit rating for the nature of the collateral, and benchmarks the borrowing rate against observable yields on comparable securities with a similar term. The incremental borrowing rate, the ROU asset and the lease liability are reevaluated upon a lease modification. It bases the ROU asset on the lease liability adjusted for any prepaid or deferred rent. The Company elected to combine lease and non-lease components for all underlying assets groups. The Company determines the lease term at the commencement date by considering whether renewal options and termination options are reasonably assured of exercise. For short term leases, the Company does not recognize a right-of-use asset and lease liability and recognizes the lease expense over the term of the lease on a straight-line basis.
Rent expense for operating leases is recognized on a straight-line basis over the lease term and is included in operating expenses on the statements of operations and comprehensive loss. The variable lease payments primarily consist of common area maintenance and other operating costs.
The Company assesses the probability of collecting lease payments under its sublease at each reporting date. The Company currently does not deem the collection of lease payments probable and therefore sublease income is constrained at the lesser of cash payments or straight-line sublease income. Sublease income/loss for operating leases is classified in general and administrative expenses based on payments collected and tenant improvement allowance disbursed to the subtenant.
Revenue Recognition
Revenue Recognition—The Company has primarily generated revenue through license, research and collaboration arrangements with its strategic partners.
The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, Revenue from Contracts with Customers, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.
Research and Development Expenses
Research and Development Expenses—Research and development expenses are charged to expense as incurred. Research and development expenses include primarily personnel-related costs, stock-based compensation expense, laboratory supplies, consulting costs, external contract research and development expenses, including expenses incurred under agreements with contract research organizations (“CROs”), the cost of acquiring, developing and manufacturing clinical trial materials, and overhead expenses, such as rent, equipment depreciation, insurance and utilities. Advance payments for goods or services for future research and development activities are deferred and expensed as the goods are delivered or the related services are performed.
The Company estimates research and clinical trial expenses based on the services performed pursuant to contracts with research institutions and clinical research organizations that conduct and manage nonclinical studies and clinical trials on the Company’s behalf. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. These estimates are based on communications with the third-party service providers and the Company’s estimates of accrued expenses and on information available at each balance sheet date. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual accordingly.
Fair Value Measurements
Fair Value Measurements—Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The carrying amounts of the Company’s financial instruments, including cash equivalents approximate their fair values due to their short-term maturities. See Note 4, Fair Value Measurements for the methodologies and assumptions used in valuing financial instruments.
Stock-based Compensation Expense
Stock-based Compensation Expense—Stock-based compensation expense related to stock awards to employees is measured at fair value of the award on the date of the grant. The Company estimates the grant-date fair value, and the resulting stock-based compensation expense, using the Black-Scholes valuation model for stock options and employee stock purchase and using intrinsic value, which is the closing price of its common stock on the date of the grant, for restricted stock units (“RSUs”) and performance stock units (“PSUs”). Expense recognition of PSU and performance-based options commences when the associated performance-based criteria are determined to be probable.
The fair value of the award that is ultimately expected to vest is recognized as expense on a straight-line basis over the requisite service period, which is generally the vesting period.
The Black-Scholes valuation model requires the use of following assumptions:
Expected Term—The expected term assumption represents the period that the Company’s stock-based awards are expected to be outstanding and is determined using the simplified method.
Expected Volatility—Expected volatility is based on the Company’s historical stock price volatility.
Expected Dividend—The Black-Scholes valuation model calls for a single expected dividend yield as an input. The Company has never paid dividends and has no plans to pay dividends.
Risk-Free Interest Rate—The risk-free interest rate is based on the U.S. Treasury zero-coupon issues in effect at the time of grant for periods corresponding with the expected term of option.
Income Taxes
Income Taxes—The Company accounts for income taxes using the asset and liability method. The Company records deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse.
In evaluating the ability to recover its deferred income tax assets, the Company considers all available positive and negative evidence, including its operating results, ongoing tax planning and forecasts of future taxable income on a jurisdiction-by-jurisdiction basis. In the event the Company determines that it would be able to realize its deferred income tax assets in the future in excess of their net recorded amount, it would make an adjustment to the valuation allowance that would reduce the provision for income taxes. Conversely, in the event that all or part of the net deferred tax assets are determined not to be realizable in the future, an adjustment to the valuation allowance would be charged to earnings in the period when such determination is made. As of December 31, 2024 and 2023, the Company has recorded a full valuation allowance on its deferred tax assets.
Tax benefits related to uncertain tax positions are recognized when it is more likely than not that a tax position will be sustained upon examination. Interest and penalties related to unrecognized tax liabilities are included within the provision for income tax.
Comprehensive Loss Comprehensive Loss—Comprehensive loss comprises net loss and other comprehensive loss. Other comprehensive loss consists of foreign currency translation adjustments and unrealized gain or loss on marketable securities.
Basic and Diluted Net Loss Per Share
Basic and Diluted Net Loss Per Share Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding for the period. Diluted net loss per share is computed by giving effect to all potential dilutive common stock equivalents outstanding for the period using the treasury stock method. Outstanding stock options, RSUs, and employee stock purchase plan (“ESPP”) are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive.
Recent Accounting Pronouncements and Recently Adopted Accounting Pronouncements
Recent Accounting Pronouncements
In November 2024, the FASB issued ASU 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (“ASU 2024-03”). ASU 2024-03 requires disaggregated disclosures of certain categories of expenses in the notes to the financial statements that are included in expense line items on the face of the income statement in annual and interim periods. ASU 2024-03 is effective for annual periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027. The Company is evaluating the impact of this guidance on its financial statements and related disclosures.
In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures (“ASU 2023-09”). ASU 2023-09 requires enhanced annual disclosures regarding the rate reconciliation and income taxes paid. ASU 2023-09 is effective for annual periods beginning after December 15, 2024 and may be adopted on a prospective or retrospective basis. Early adoption is permitted. The Company is evaluating the impact of this guidance on its financial statements and related disclosures.
In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures (“ASU 2023-07”). The amendments in ASU 2023-07 are intended to improve reportable segment disclosure, primarily through enhanced disclosures about significant segment expenses. ASU 2023-07 is effective for annual periods beginning after December 15, 2023, and interim periods beginning after December 15, 2024. The amendments in this ASU should be applied retrospectively to all prior periods presented in the financial statements. The Company retrospectively adopted ASU 2023-07 on December 31, 2024. The adoption of ASU 2023-07 did not have a material financial impact on the Company’s consolidated financial statements.
v3.25.1
Restatement of Previously Issued Financial Statements (Tables)
12 Months Ended
Dec. 31, 2024
Accounting Changes and Error Corrections [Abstract]  
Restatement of Previously Issued Financial Statements
Consolidated Balance Sheet
As of December 31, 2023
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$75,000 $— $75,000 
Short-term investments21,526 — 21,526 
Prepaid expenses and other current assets6,247 6,247 
Total current assets102,773 — 102,773 
Operating lease right-of-use assets52,266 (17,242)
(a) (c)
35,024 
Property and equipment, net14,764 — 14,764 
Restricted cash1,976 — 1,976 
Deposit and other long-term assets1,231 1,231 
Total assets$173,010 $(17,242)$155,768 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable$1,921 $— $1,921 
Accrued expenses and other current liabilities12,584 — 12,584 
Lease liability, current portion10,409 (4,933)
(a)
5,476 
Total current liabilities24,914 (4,933)19,981 
Long-term liabilities:
Lease liability, net of current portion64,627 3,938 
(a) (c)
68,565 
Total liabilities89,541 (995)88,546 
Commitments and contingencies
Stockholders’ equity:
Preferred stock, $0.0001 par value, 5,000 shares authorized; no shares issued and outstanding
— — — 
Common stock, $0.0001 par value, 300,000 shares authorized at December 31, 2023: 10,143 shares issued and outstanding at December 31, 2023
— 
Additional paid-in capital1,003,718 — 1,003,718 
Accumulated other comprehensive loss(473)— (473)
Accumulated deficit(919,777)(16,247)
(a) (b)
(936,024)
Total stockholders’ equity83,469 (16,247)67,222 
Total liabilities and stockholders’ equity$173,010 $(17,242)$155,768 
Consolidated Statement of Operations and Comprehensive Loss
Year ended December 31, 2023
As Previously Reported
Adjustment
Reference
As Restated
License revenue$3,600 $— $3,600 
Operating expenses:
Research and development77,676 (190)
(b)
77,486 
General and administrative49,915 5,141 
(a)
55,056 
Total operating expenses127,591 4,951 132,542 
Operating loss(123,991)(4,951)(128,942)
Other income, net5,748 — 5,748 
Net loss before income taxes(118,243)(4,951)(123,194)
Income tax benefit
1,078 — 1,078 
Net loss$(117,165)$(4,951)$(122,116)
Other comprehensive gain:
Net unrealized gain on marketable securities
1,057 — 1,057 
Foreign currency translation adjustment— 
Comprehensive loss$(116,107)$(4,951)$(121,058)
Net loss per share - basic and diluted$(11.62)$— $(12.11)
Weighted-average common shares outstanding - basic and diluted10,082 — 10,082 
Consolidated Statement of Cash Flows
Year ended December 31, 2023
As Previously Reported
Adjustment
Reference
As Restated
Cash flows from operating activities:
Net loss$(117,165)$(4,951)
(a) (b)
$(122,116)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense13,027 5,230 
(a)
18,257 
Non-cash sublease income
— (89)(89)
Changes in operating assets and liabilities:
Accrued expenses and other liabilities(5,206)(190)
(b)
(5,396)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets and liabilities$13,711 $(6,079)
(a) (c)
$7,632 
v3.25.1
Fair Value Measurements and Fair Value of Financial Instruments (Tables)
12 Months Ended
Dec. 31, 2024
Fair Value Disclosures [Abstract]  
Schedule of Cash Equivalents and Short-term Investments
The following is a summary of the Company’s cash equivalents and short-term investments (in thousands):
December 31, 2024
Amortized
Cost Basis
Unrealized
Gains
Unrealized
Losses
Estimated
Fair Value
Level 1
Money market funds$165 $— $— $165 
Level 2
Commercial paper78,500 10 (9)78,501 
U.S. government and agency securities41,210 56 — 41,266 
Total cash equivalents and short-term investments119,875 66 (9)119,932 
Less: Cash equivalents(54,901)— (54,893)
Total short-term investments$64,974 $66 $(1)$65,039 
December 31, 2023
Amortized
Cost Basis
Unrealized
Gains
Unrealized
Losses
Estimated
Fair Value
Level 1
Money market funds$10,204 $— $— $10,204 
Level 2
Commercial paper64,693 — (35)64,658 
U.S. government and agency securities17,616 (17)17,603 
Total cash equivalents and short-term investments92,513 (52)92,465 
Less: Cash equivalents(70,972)— 33 (70,939)
Total short-term investments$21,541 $$(19)$21,526 
v3.25.1
Leases (Tables)
12 Months Ended
Dec. 31, 2024
Leases [Abstract]  
Schedule of Undiscounted Future Lease Payments under Operating Lease
The following table summarizes the undiscounted future non-cancellable lease payments under the lease agreements as of December 31, 2024 (in thousands):
December 31,Operating Leases
2025$12,142 
202614,892 
202715,278 
202815,677 
202916,089 
Thereafter93,087 
Total undiscounted lease payments$167,165 
Less: Imputed Interest
(75,460)
Total$91,705 
v3.25.1
Balance Sheet Components (Tables)
12 Months Ended
Dec. 31, 2024
Balance Sheet Related Disclosures [Abstract]  
Schedule of Property and Equipment, Net
Property and equipment, net consisted of the following:
December 31,
20242023
(In thousands)
Laboratory equipment$14,963 $14,638 
Leasehold improvements13,779 13,586 
Computer equipment and software901 868 
Construction in progress184 
Total property and equipment29,652 29,276 
Less accumulated depreciation and amortization(18,045)(14,512)
Property and equipment, net$11,607 $14,764 
Schedule of Accrued Expenses and Other Current Liabilities
Accrued expenses and other current liabilities consist of the following:
December 31,
20242023
(In thousands)
Employee compensation$9,540 $8,040 
Accrued nonclinical, clinical and process development costs4,401 3,367 
Accrued professional fees554 351 
State income tax payable123 101 
Other1,002 725 
Total accrued expenses and other current liabilities$15,620 $12,584 
v3.25.1
Stockholders' Equity (Tables)
12 Months Ended
Dec. 31, 2024
Share-Based Payment Arrangement [Abstract]  
Schedule of Stock Options Activity
The following table summarizes stock option activity under the Company’s stock plans and related information:
(In thousands, except exercise prices and years)Options
Outstanding
Weighted-
Average
Exercise
Price
Weighted-
Average
Remaining Contract
Life (in years)
Aggregate
Intrinsic Value (a)
Balance at December 31, 20232,303 $48.70 6.8$99 
Granted1,971 10.39 
Exercised(15)9.97 
Canceled/forfeited(350)50.28 
Balance at December 31, 20243,909 $29.39 8.1$— 
Vested and expected to vest as of December 31, 20243,909 $29.39 8.1$— 
Exercisable at December 31, 20241,504 $58.95 6.5$— 
(a)The aggregate intrinsic value is calculated as the difference between the stock option exercise price and the closing price of the Company’s common stock as quoted on a national exchange.
Schedule of Fair Value of Option Issued Valuation Assumptions
The fair value of each stock option issued was estimated at the date of grant using the Black-Scholes valuation model with the following weighted-average assumptions:
OptionsEmployee Stock Purchase Plan
Years ended December 31,Years ended December 31,
2024202320242023
Expected volatility95%90%83%78%
Expected term (in years)6.06.01.31.3
Expected dividend yield
Risk-free interest rate3.9%4.2%4.3%5.0%
Schedule of Restricted Stock Units Activity
The following table summarizes the RSU activity under the Company’s stock plans and related information:
(In thousands, except grant date fair value and years)Number of
Units
Weighted-
Average
Grant Date
Fair Value
Weighted-
Average
Remaining
Contractual Term
(in years)
Balance at December 31, 2023154 21.73 1.4
Granted160 12.04 
Vested and released(29)26.20 
Forfeited(26)14.22 
Balance at December 31, 2024259 $16.00 1.2
Schedule of Stock-based Compensation Expense
The following table presents the Company’s stock-based compensation expense:
Years ended December 31,
20242023
(In thousands)
Research and development$4,544 $4,969 
General and administrative9,806 12,600 
Total share-based compensation expense$14,350 $17,569 
v3.25.1
Segment Reporting (Tables)
12 Months Ended
Dec. 31, 2024
Segment Reporting [Abstract]  
Schedule of Segment Reporting Information, by Segment
The following table presents the segment loss, including significant segment expenses, for the years ended December 31, 2024 and 2023 (in thousands):
Years ended December 31,
20242023 As Restated
(In thousands)
License Revenue$1,000 $3,600 
Less:
Compensation and benefits expenses
56,546 54,572 
Clinical trial and manufacturing
22,333 20,235 
Facilities and IT
37,841 39,114 
Other research and development expense
4,728 4,985 
Other segment expenses(a)
18,711 13,636 
Total operating expenses140,159 132,542 
Operating loss(139,159)(128,942)
Other income, net
8,232 5,748 
Net loss before income taxes(130,927)(123,194)
Income tax benefit (provision)— 1,078 
Segment and consolidated net loss$(130,927)$(122,116)
(a)
Other segment expenses include professional services, consultants and contractors, travel and entertainment expenses, and general business expenses.
v3.25.1
Income Taxes (Tables)
12 Months Ended
Dec. 31, 2024
Income Tax Disclosure [Abstract]  
Schedule of Components of Loss Before Income Taxes
The following table presents domestic and foreign components of loss before provision for income taxes:
Years ended December 31,
20242023 As Restated
(In thousands)
U.S.$(130,768)$(123,040)
Foreign(159)(154)
Loss before income taxes$(130,927)$(123,194)
Schedule of Components of Income Tax (Benefit) Provision
The components of the Company’s income tax (benefit) provision were as follows:
Years ended December 31,
20242023 As Restated
(In thousands)
Current:
Foreign$— $(1,078)
Total current tax (benefit) provision— (1,078)
Deferred
Foreign— — 
Total deferred tax provision— — 
Total income tax (benefit) provision$— $(1,078)
Schedule of Reconciliation of Income Tax Expense Computed Statutory Federal Income Tax and Financial Statements
The income tax provision for the years ended December 31, 2024 and 2023 differed from the amounts computed by applying the statutory federal income tax rate of 21% to pretax loss as a result of the following:
Years ended December 31,
20242023 As Restated
(In thousands)
Federal income tax benefit at statutory rate$(27,495)$(25,871)
Stock compensation3,189 6,001 
Non-deductible expenses41 21 
Research and development tax credits(5,642)(1,810)
Change in valuation allowance23,801 19,558 
Foreign rate differential(14)(14)
Impact of internal reorganization— 1,323 
Uncertain tax positions6,100 (1,078)
Other20 792 
Total tax (benefit) provision$— $(1,078)
Schedule of Deferred Tax Assets The following table presents significant components of the Company’s deferred tax assets and liabilities:
As of December 31,
20242023 As Restated
(In thousands)
Deferred tax assets:
Net operating loss carryforwards$137,140 $128,171 
Accruals, reserve and other1,972 1,749 
Tax credit carryforwards29,263 21,254 
Stock-based compensation6,349 7,274 
Intangibles11 
Lease obligation19,622 17,322 
Capital losses9,850 9,850 
Section 174 R&D capitalization35,842 29,688 
Other
Total deferred tax assets before valuation allowance240,050 215,322 
Valuation allowance(232,058)(205,861)
Total deferred tax assets7,992 9,461 
Deferred tax liabilities:
Right-of-use assets(7,149)(8,153)
Property and equipment(843)(1,308)
Total deferred tax liabilities$(7,992)$(9,461)
Net deferred tax assets$— $— 
Schedule of Unrecognized Tax Benefits A reconciliation of the unrecognized tax benefits is as follows:
Years ended December 31,
20242023 As Restated
(In thousands)
Unrecognized tax benefits as of the beginning of the year$22,758 $23,069 
Increase related to tax positions taken during the prior year— 43 
Decrease related to tax positions taken during the prior year
(2,880)— 
Increase related to tax position taken during the current year
7,310 3,121 
Decrease related to tax position taken during the current year— (3,475)
Unrecognized tax benefits as of the end of the year$27,188 $22,758 
v3.25.1
Net Loss per Share (Tables)
12 Months Ended
Dec. 31, 2024
Earnings Per Share [Abstract]  
Schedule of Antidilutive Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share
The following common stock equivalents outstanding at the end of the periods presented were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect:
As of December 31,
20242023
(In thousands)
Stock options3,909 2,303 
Restricted stock units259 154 
ESPP32 17 
4,200 2,474 
v3.25.1
Quarterly Financial Information (Unaudited) (Tables)
12 Months Ended
Dec. 31, 2024
Quarterly Financial Information Disclosure [Abstract]  
Schedule of Quarterly Financial Information
The unaudited interim financial statements reflect all adjustments which are, in the opinion of management, necessary for a fair statement of the results for the interim periods presented (in thousands, except per share data).
Condensed Consolidated Balance Sheets
September 30, 2024 (unaudited)
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$92,851 $— $92,851 
Short-term investments
60,390 — 60,390 
Prepaid expenses and other current assets7,839 — 7,839 
Total current assets161,080 — 161,080 
Operating lease right-of-use assets50,695 (17,015)
(a) (d)
33,680 
Property and equipment, net
12,215 — 12,215 
Deferred rent receivable
7,116 (7,116)
(b)
— 
Restricted cash
1,976 — 1,976 
Deposit and other long-term assets
1,293 — 1,293 
Total assets$234,375 $(24,131)$210,244 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
$2,110 — $2,110 
Accrued expenses and other current liabilities14,886 — 14,886 
Lease liability, current portion
10,661 (5,044)
(a)
5,617 
Total current liabilities27,657 (5,044)22,613 
Long-term liabilities:
Lease liability, net of current portion62,602 16,759 
(a) (d)
79,361 
Total liabilities90,259 11,715 101,974 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital1,134,556 — 1,134,556 
Accumulated other comprehensive loss(268)— (268)
Accumulated deficit(990,174)(35,846)
(a) (b)
(1,026,020)
Total stockholders’ equity144,116 (35,846)108,270 
Total liabilities and stockholders’ equity$234,375 $(24,131)$210,244 
June 30, 2024 (unaudited)
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$127,321 $— $127,321 
Short-term investments
46,506 — 46,506 
Prepaid expenses and other current assets5,591 — 5,591 
Total current assets179,418 — 179,418 
Operating lease right-of-use assets51,237 (17,241)
(a) (d)
33,996 
Property and equipment, net
13,047 13,047 
Deferred rent receivable
6,963 (6,963)
(b)
— 
Restricted cash
1,976 — 1,976 
Deposit and other long-term assets
1,162 — 1,162 
Total assets$253,803 $(24,204)$229,599 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
1,973 — 1,973 
Accrued expenses and other current liabilities9,912 — 9,912 
Lease liability, current portion
10,576 (5,006)
(a)
5,570 
Total current liabilities22,461 (5,006)17,455 
Long-term liabilities:
Lease liability, net of current portion63,313 11,431 
(a) (d)
74,744 
Total liabilities85,774 6,425 92,199 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital1,131,592 — 1,131,592 
Accumulated other comprehensive loss(525)— (525)
Accumulated deficit(963,040)(30,629)
(a) (b)
(993,669)
Total stockholders’ equity168,029 (30,629)137,400 
Total liabilities and stockholders’ equity$253,803 $(24,204)$229,599 
March 31, 2024 (unaudited)
As Previously Reported
Adjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$149,608 $— $149,608 
Short-term investments
43,720 — 43,720 
Prepaid expenses and other current assets5,802 — 5,802 
Total current assets199,130 — 199,130 
Operating lease right-of-use assets51,760 (17,282)
(a) (d)
34,478 
Property and equipment, net
13,766 — 13,766 
Restricted cash
1,976 — 1,976 
Deposit and other long-term assets
1,196 — 1,196 
Total assets$267,828 $(17,282)$250,546 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
1,897 — 1,897 
Accrued expenses and other current liabilities9,160 — 9,160 
Lease liability, current portion
10,492 (4,971)
(a)
5,521 
Total current liabilities21,549 (4,971)16,578 
Long-term liabilities:
Lease liability, net of current portion63,991 6,296 
(a) (d)
70,287 
Total liabilities85,540 1,325 86,865 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital1,127,383 — 1,127,383 
Accumulated other comprehensive loss(533)— (533)
Accumulated deficit(944,564)(18,607)
(a)
(963,171)
Total stockholders’ equity182,288 (18,607)163,681 
Total liabilities and stockholders’ equity$267,828 $(17,282)$250,546 
September 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$105,366 $— $105,366 
Short-term investments
11,716 — 11,716 
Prepaid expenses and other current assets7,514 — 7,514 
Total current assets124,596 — 124,596 
Operating lease right-of-use assets52,757 (17,158)
(a) (d)
35,599 
Property and equipment, net
15,497 — 15,497 
Restricted cash
2,650 — 2,650 
Deposit and other long-term assets
1,270 — 1,270 
Total assets$196,770 $(17,158)$179,612 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
2,170 — 2,170 
Accrued expenses and other current liabilities16,362 — 16,362 
Lease liability, current portion
10,324 (4,897)
(a)
5,427 
Total current liabilities28,856 (4,897)23,959 
Long-term liabilities:
Lease liability, net of current portion65,200 3,196 
(a) (d)
68,396 
Total liabilities94,056 (1,701)92,355 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital999,328 — 999,328 
Accumulated other comprehensive loss(552)— (552)
Accumulated deficit(896,063)(15,457)
(a) (c)
(911,520)
Total stockholders’ equity102,714 (15,457)87,257 
Total liabilities and stockholders’ equity$196,770 $(17,158)$179,612 
June 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$111,187 $— $111,187 
Short-term investments
30,266 — 30,266 
Prepaid expenses and other current assets5,690 — 5,690 
Total current assets147,143 — 147,143 
Operating lease right-of-use assets58,287 (17,054)
(a) (d)
41,233 
Property and equipment, net
32,041 — 32,041 
Restricted cash
2,650 — 2,650 
Deposit and other long-term assets
1,308 — 1,308 
Total assets$241,429 $(17,054)$224,375 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
1,418 — 1,418 
Accrued expenses and other current liabilities17,704 — 17,704 
Lease liability, current portion
25,396 (4,861)
(a)
20,535 
Total current liabilities44,518 (4,861)39,657 
Long-term liabilities:
Lease liability, net of current portion65,756 2,734 
(a) (d)
68,490 
Total liabilities110,274 (2,127)108,147 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital994,938 — 994,938 
Accumulated other comprehensive loss(606)— (606)
Accumulated deficit(863,178)(14,927)
(a) (c)
(878,105)
Total stockholders’ equity131,155 (14,927)116,228 
Total liabilities and stockholders’ equity$241,429 $(17,054)$224,375 
March 31, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Assets
Current assets:
Cash and cash equivalents$67,552 $— $67,552 
Short-term investments
96,733 — 96,733 
Prepaid expenses and other current assets4,588 — 4,588 
Total current assets168,873 — 168,873 
Operating lease right-of-use assets69,503 (22,905)
(a) (d)
46,598 
Property and equipment, net
33,347 — 33,347 
Restricted cash
2,503 — 2,503 
Deposit and other long-term assets
1,351 — 1,351 
Total assets$275,577 $(22,905)$252,672 
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable
2,863 — 2,863 
Accrued expenses and other current liabilities17,344 — 17,344 
Lease liability, current portion
26,265 (5,531)
(a)
20,734 
Total current liabilities46,472 (5,531)40,941 
Long-term liabilities:
Lease liability, net of current portion71,348 (3,462)
(a) (d)
67,886 
Total liabilities117,820 (8,993)108,827 
Commitments and contingencies
Stockholders’ equity:
Preferred stock— — — 
Common stock
— 
Additional paid-in capital990,223 — 990,223 
Accumulated other comprehensive loss(799)— (799)
Accumulated deficit(831,668)(13,912)
(a) (c)
(845,580)
Total stockholders’ equity157,757 (13,912)143,845 
Total liabilities and stockholders’ equity$275,577 (22,905)$252,672 
Quarterly and Year to Date Condensed Consolidated Statements of Operations and Comprehensive Loss
Three Months Ended
Nine Months Ended
September 30, 2024 (unaudited)September 30, 2024 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
License revenue$1,000 $— $1,000 $1,000 $— $1,000 
Operating expenses:
Research and development20,439 — 20,439 52,946 — 52,946 
General and administrative9,782 5,217 
(a)
14,999 24,996 19,599 
(a) (b)
44,595 
Total operating expenses30,221 5,217 35,438 77,942 19,599 97,541 
Operating loss(29,221)(5,217)(34,438)(76,942)(19,599)(96,541)
Other income, net2,087— 2,0876,545— 6,545
Net loss(27,134)(5,217)(32,351)(70,397)(19,599)(89,996)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities241 — 241 198 — 198 
Foreign currency translation adjustment16 — 16 — 
Comprehensive loss$(26,877)$(5,217)$(32,094)$(70,192)$(19,599)$(89,791)
Net loss per share — basic and diluted$(1.30)— $(1.55)$(3.63)— $(4.64)
Weighted-average common shares used to compute net loss per share - basic and diluted20,876 — 20,876 19,408 — 19,408 

Three Months Ended
Six Months Ended
June 30, 2024 (unaudited)June 30, 2024 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
Operating expenses:
Research and development$17,097 $— $17,097 $32,507 $— $32,507 
General and administrative3,785 12,022 
(a) (b)
15,807 15,214 14,382 
(a) (b)
29,596 
Total operating expenses20,882 12,022 32,904 47,721 14,382 62,103 
Operating loss(20,882)(12,022)(32,904)(47,721)(14,382)(62,103)
Other income, net2,406 — 2,406 4,458 — 4,458 
Net loss(18,476)(12,022)(30,498)(43,263)(14,382)(57,645)
Other comprehensive gain (loss):
Net unrealized loss on marketable securities
(2)— (2)(43)— (43)
Foreign currency translation adjustment10 — 10 (9)— (9)
Comprehensive loss$(18,468)(12,022)$(30,490)$(43,315)$(14,382)$(57,697)
Net loss per share — basic and diluted$(0.89)— $(1.46)$(2.31)— $(3.08)
Weighted-average common shares used to compute net loss per share - basic and diluted20,852 — 20,852 18,713  18,713 
Three Months Ended
March 31, 2024 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
Operating expenses:
Research and development$15,410 $— $15,410 
General and administrative11,429 2,360 
(a)
13,789 
Total operating expenses26,839 2,360 29,199 
Operating loss(26,839)(2,360)(29,199)
Other income, net2,052 — 2,052 
Net loss(24,787)(2,360)(27,147)
Other comprehensive gain (loss):
Net unrealized loss on marketable securities
(41)— (41)
Foreign currency translation adjustment(19)— (19)
Comprehensive loss$(24,847)$(2,360)$(27,207)
Net loss per share — basic and diluted$(1.50)— $(1.65)
Weighted-average common shares used to compute net loss per share - basic and diluted16,479 — 16,479 

Three Months EndedNine Months Ended
September 30, 2023 (unaudited)September 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
License revenue$— $— $— $3,600 $— $3,600 
Operating expenses:
Research and development20,740 — 20,740 62,398 (190)
(c)
62,208 
General and administrative13,789 530 
(a)
14,319 39,035 4,351 
(a)
43,386 
Total operating expenses34,529 530 35,059 101,433 4,161 105,594 
Operating loss(34,529)(530)(35,059)(97,833)(4,161)(101,994)
Other income, net1,661— 1,6614,437— 4,437
Net loss before income taxes(32,868)(530)(33,398)(93,396)(4,161)(97,557)
Income tax provision(17)(17)(55)(55)
Net loss(32,885)(530)(33,415)(93,451)(4,161)(97,612)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities66 — 66 1,003 — 1,003 
Foreign currency translation adjustment(12)— (12)(24)— (24)
Comprehensive loss$(32,831)(530)$(33,361)$(92,472)$(4,161)$(96,633)
Net loss per share — basic and diluted$(3.26)— $(3.31)$(9.28)— $(9.69)
Weighted-average common shares used to compute net loss per share - basic and diluted10,099 — 10,099 10,069 — 10,069 
Three Months Ended
Six Months Ended
June 30, 2023 (unaudited)June 30, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As RestatedAs Previously ReportedAdjustment
Reference
As Restated
License revenue$— $— $— $3,600 $— $3,600 
Operating expenses:
Research and development$20,599 $— $20,599 $41,658 $(190)
(c)
$41,468 
General and administrative12,466 1,015 
(a)
13,481 25,246 3,821 
(a)
29,067 
Total operating expenses33,065 1,015 34,080 66,904 3,631 70,535 
Operating loss(33,065)(1,015)(34,080)(63,304)(3,631)(66,935)
Other income, net1,576 — 1,576 2,776 — 2,776 
Net loss before income taxes(31,489)(1,015)(32,504)(60,528)(3,631)(64,159)
Income tax provision(21)(21)(38)(38)
Net loss(31,510)(1,015)(32,525)(60,566)(3,631)(64,197)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities
198 — 198 937 — 937 
Foreign currency translation adjustment(5)— (5)(12)— (12)
Comprehensive loss$(31,317)(1,015)$(32,332)$(59,641)$(3,631)$(63,272)
Net loss per share — basic and diluted$(3.13)— $(3.23)$(6.02)— $(6.39)
Weighted-average common shares used to compute net loss per share - basic and diluted10,076 — 10,076 10,053 — 10,053 

Three Months Ended
March 31, 2023 (unaudited)
As Previously ReportedAdjustment
Reference
As Restated
License revenue$3,600 $— $3,600 
Operating expenses:
Research and development$21,059 $(190)
(c)
$20,869 
General and administrative12,780 2,806 
(a)
15,586 
Total operating expenses33,839 2,616 36,455 
Operating loss(30,239)(2,616)(32,855)
Other income, net1,200 — 1,200 
Net loss before income taxes(29,039)(2,616)(31,655)
Income tax provision(17)— (17)
Net loss(29,056)(2,616)(31,672)
Other comprehensive gain (loss):
Net unrealized gain on marketable securities
739 — 739 
Foreign currency translation adjustment(7)— (7)
Comprehensive loss$(28,324)(2,616)$(30,940)
Net loss per share — basic and diluted$(2.90)— $(3.16)
Weighted-average common shares used to compute net loss per share - basic and diluted
10,030 — 10,030 
Condensed Consolidated Statements of Cash Flows
The effects of this restatement on certain line items of the condensed consolidated statements of cash flows are summarized in the following tables (in thousands). There was no impact on net cash used in operating activities or within any line items within investing and financing activities.
Nine Months Ended September 30, 2024
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(70,397)$(19,599)
(a) (b)
$(89,996)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense1,571 4,006 
(a)
5,577 
Non-cash sublease loss
— 8,477 
(a)
8,477 
Changes in operating assets and liabilities:
Deposit and other long-term assets and deferred rent receivable(7,178)7,116 
(b)
(62)

Six Months Ended June 30, 2024
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(43,263)$(14,382)
(a) (b)
$(57,645)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense1,029 2,654 
(a)
3,683 
Non-cash sublease loss
— 4,765 
(a)
4,765 
Changes in operating assets and liabilities:
Deposit and other long-term assets and deferred rent receivable(6,894)6,963 
(b)
69 

Three Months Ended March 31, 2024
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(24,787)$(2,360)
(a)
$(27,147)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense506 1,314 
(a)
1,820 
Non-cash sublease loss
— 1,046 
(a)
1,046 
Nine Months Ended September 30, 2023
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(93,451)$(4,161)
(a) (c)
$(97,612)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense12,466 3,916 
(a)
16,382 
Non-cash sublease loss
— 435 
(a)
435 
Changes in operating assets and liabilities:— 
Accrued expenses and other liabilities(1,452)(190)
(c)
(1,642)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets
$13,711 $(6,079)$7,632 

Six Months Ended June 30, 2023
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(60,566)$(3,631)
(a) (c)
(64,197)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense6,936 2,602 
(a)
9,538 
Non-cash sublease loss
— 1,219 
(a)
1,219 
Changes in operating assets and liabilities:
Accrued expenses and other liabilities(525)(190)
(c)
(715)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets
$13,711 $(6,079)$7,632 
Three Months Ended March 31, 2023
As Previously Reported
Adjustments
Reference
As Restated
Cash flows from operating activities:
Net loss$(29,056)$(2,616)
(a) (c)
$(31,672)
Adjustments to reconcile net loss to net cash used in operating activities:
Non-cash lease expense1,427 1,239 
(a)
2,666 
Non-cash sublease loss
— 1,567 1,567 
Changes in operating assets and liabilities:— 
Accrued expenses and other liabilities(749)(190)
(c)
(939)
Supplemental schedule of noncash information
Remeasurement of operating lease right-of-use assets
$8,004 $(104)$7,900 
v3.25.1
Description of the Business - Additional Information (Details) - USD ($)
$ in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]                
Accumulated deficit $ 1,066,951 $ 936,024 $ 1,026,020 $ 993,669 $ 963,171 $ 911,520 $ 878,105 $ 845,580
Cash used in operations 92,462 $ 90,902            
Cash, cash equivalents, and short-term investments $ 125,700              
v3.25.1
Restatement of Previously Issued Financial Statements (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Interim Period, Costs Not Allocable [Line Items]                          
Research and development expense reversal $ (20,439) $ (17,097) $ (15,410) $ (20,740) $ (20,599) $ (20,869) $ (32,507) $ (41,468) $ (52,946) $ (62,208) $ (77,041) $ (77,486)  
Right-to-use asset 33,680 33,996 34,478 35,599 41,233 46,598 33,996 41,233 33,680 35,599 33,611 35,024  
Lease liability                     91,705    
Accumulated deficit 1,026,020 993,669 963,171 911,520 878,105 845,580 993,669 878,105 1,026,020 911,520 $ 1,066,951 936,024  
Adjustment                          
Interim Period, Costs Not Allocable [Line Items]                          
Research and development expense reversal 0 0 0 0 0 190 0 190 0 190   190  
Right-to-use asset (17,015) (17,241) (17,282) (17,158) (17,054) (22,905) (17,241) (17,054) (17,015) (17,158)   (17,242)  
Accumulated deficit $ 35,846 $ 30,629 $ 18,607 $ 15,457 $ 14,927 13,912 $ 30,629 $ 14,927 $ 35,846 $ 15,457   $ 16,247 $ 11,300
Adjustment | Redwood, California Facility Lease                          
Interim Period, Costs Not Allocable [Line Items]                          
Right-to-use asset           300              
Lease liability           $ 300              
v3.25.1
Restatement of Previously Issued Financial Statements - Consolidated Balance Sheet (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Dec. 31, 2023
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Dec. 31, 2022
Current assets:                  
Cash and cash equivalents $ 60,652 $ 92,851 $ 127,321 $ 149,608 $ 75,000 $ 105,366 $ 111,187 $ 67,552  
Short-term investments 65,039 60,390 46,506 43,720 21,526 11,716 30,266 96,733  
Prepaid expenses and other current assets 5,609 7,839 5,591 5,802 6,247 7,514 5,690 4,588  
Total current assets 131,300 161,080 179,418 199,130 102,773 124,596 147,143 168,873  
Operating lease right-of-use assets 33,611 33,680 33,996 34,478 35,024 35,599 41,233 46,598  
Property and equipment, net 11,607 12,215 13,047 13,766 14,764 15,497 32,041 33,347  
Restricted cash 1,976 1,976 1,976 1,976 1,976 2,650 2,650 2,503  
Deposit and other long-term assets 1,347 1,293 1,162 1,196 1,231 1,270 1,308 1,351  
Total assets 179,841 210,244 229,599 250,546 155,768 179,612 224,375 252,672  
Current liabilities:                  
Accounts payable 1,610 2,110 1,973 1,897 1,921 2,170 1,418 2,863  
Accrued expenses and other current liabilities 15,620 14,886 9,912 9,160 12,584 16,362 17,704 17,344  
Lease liability, current portion 5,668 5,617 5,570 5,521 5,476 5,427 20,535 20,734  
Total current liabilities 22,898 22,613 17,455 16,578 19,981 23,959 39,657 40,941  
Long-term liabilities:                  
Lease liability, net of current portion 86,037 79,361 74,744 70,287 68,565 68,396 68,490 67,886  
Total liabilities 109,127 101,974 92,199 86,865 88,546 92,355 108,147 108,827  
Commitments and contingencies  
Stockholders’ equity:                  
Preferred stock, $0.0001 par value, 5,000 shares authorized; no shares issued and outstanding 0 0 0 0 0 0 0 0  
Common stock, $0.0001 par value, 300,000 shares authorized at December 31, 2024: 20,848 and 10,143 shares issued and outstanding at December 31, 2024 and 2023, respectively 2 2 2 2 1 1 1 1  
Additional paid-in capital 1,138,070 1,134,556 1,131,592 1,127,383 1,003,718 999,328 994,938 990,223  
Accumulated other comprehensive loss (407) (268) (525) (533) (473) (552) (606) (799)  
Accumulated deficit (1,066,951) (1,026,020) (993,669) (963,171) (936,024) (911,520) (878,105) (845,580)  
Total stockholders’ equity 70,714 108,270 137,400 163,681 67,222 87,257 116,228 143,845 $ 170,222
Total liabilities and stockholders’ equity $ 179,841 210,244 229,599 250,546 155,768 179,612 224,375 252,672  
As Previously Reported                  
Current assets:                  
Cash and cash equivalents   92,851 127,321 149,608 75,000 105,366 111,187 67,552  
Short-term investments   60,390 46,506 43,720 21,526 11,716 30,266 96,733  
Prepaid expenses and other current assets   7,839 5,591 5,802 6,247 7,514 5,690 4,588  
Total current assets   161,080 179,418 199,130 102,773 124,596 147,143 168,873  
Operating lease right-of-use assets   50,695 51,237 51,760 52,266 52,757 58,287 69,503  
Property and equipment, net   12,215 13,047 13,766 14,764 15,497 32,041 33,347  
Restricted cash   1,976 1,976 1,976 1,976 2,650 2,650 2,503  
Deposit and other long-term assets   1,293 1,162 1,196 1,231 1,270 1,308 1,351  
Total assets   234,375 253,803 267,828 173,010 196,770 241,429 275,577  
Current liabilities:                  
Accounts payable   2,110 1,973 1,897 1,921 2,170 1,418 2,863  
Accrued expenses and other current liabilities   14,886 9,912 9,160 12,584 16,362 17,704 17,344  
Lease liability, current portion   10,661 10,576 10,492 10,409 10,324 25,396 26,265  
Total current liabilities   27,657 22,461 21,549 24,914 28,856 44,518 46,472  
Long-term liabilities:                  
Lease liability, net of current portion   62,602 63,313 63,991 64,627 65,200 65,756 71,348  
Total liabilities   90,259 85,774 85,540 89,541 94,056 110,274 117,820  
Commitments and contingencies    
Stockholders’ equity:                  
Preferred stock, $0.0001 par value, 5,000 shares authorized; no shares issued and outstanding   0 0 0 0 0 0 0  
Common stock, $0.0001 par value, 300,000 shares authorized at December 31, 2024: 20,848 and 10,143 shares issued and outstanding at December 31, 2024 and 2023, respectively   2 2 2 1 1 1 1  
Additional paid-in capital   1,134,556 1,131,592 1,127,383 1,003,718 999,328 994,938 990,223  
Accumulated other comprehensive loss   (268) (525) (533) (473) (552) (606) (799)  
Accumulated deficit   (990,174) (963,040) (944,564) (919,777) (896,063) (863,178) (831,668)  
Total stockholders’ equity   144,116 168,029 182,288 83,469 102,714 131,155 157,757  
Total liabilities and stockholders’ equity   234,375 253,803 267,828 173,010 196,770 241,429 275,577  
Adjustment                  
Current assets:                  
Cash and cash equivalents   0 0 0 0 0 0 0  
Short-term investments   0 0 0 0 0 0 0  
Prepaid expenses and other current assets   0 0 0 0 0 0  
Total current assets   0 0 0 0 0 0 0  
Operating lease right-of-use assets   (17,015) (17,241) (17,282) (17,242) (17,158) (17,054) (22,905)  
Property and equipment, net   0 0 0 0 0 0  
Restricted cash   0 0 0 0 0 0 0  
Deposit and other long-term assets   0 0 0 0 0 0  
Total assets   (24,131) (24,204) (17,282) (17,242) (17,158) (17,054) (22,905)  
Current liabilities:                  
Accounts payable   0 0 0 0 0 0 0  
Accrued expenses and other current liabilities   0 0 0 0 0 0 0  
Lease liability, current portion   (5,044) (5,006) (4,971) (4,933) (4,897) (4,861) (5,531)  
Total current liabilities   (5,044) (5,006) (4,971) (4,933) (4,897) (4,861) (5,531)  
Long-term liabilities:                  
Lease liability, net of current portion   16,759 11,431 6,296 3,938 3,196 2,734 (3,462)  
Total liabilities   11,715 6,425 1,325 (995) (1,701) (2,127) (8,993)  
Commitments and contingencies    
Stockholders’ equity:                  
Preferred stock, $0.0001 par value, 5,000 shares authorized; no shares issued and outstanding   0 0 0 0 0 0 0  
Common stock, $0.0001 par value, 300,000 shares authorized at December 31, 2024: 20,848 and 10,143 shares issued and outstanding at December 31, 2024 and 2023, respectively   0 0 0 0 0 0 0  
Additional paid-in capital   0 0 0 0 0 0 0  
Accumulated other comprehensive loss   0 0 0 0 0 0 0  
Accumulated deficit   (35,846) (30,629) (18,607) (16,247) (15,457) (14,927) (13,912) $ (11,300)
Total stockholders’ equity   (35,846) (30,629) (18,607) (16,247) (15,457) (14,927) (13,912)  
Total liabilities and stockholders’ equity   $ (24,131) $ (24,204) $ (17,282) $ (17,242) $ (17,158) $ (17,054) $ (22,905)  
v3.25.1
Restatement of Previously Issued Financial Statements - Balance Sheet Additional Items (Details) - $ / shares
Dec. 31, 2024
Dec. 31, 2023
Accounting Changes and Error Corrections [Abstract]    
Preferred stock, par value (in USD per share) $ 0.0001 $ 0.0001
Preferred stock, shares authorized (in shares) 5,000,000 5,000,000
Preferred stock, shares issued (in shares) 0 0
Preferred stock, shares outstanding (in shares) 0 0
Common stock, par value (in USD per share) $ 0.0001 $ 0.0001
Common stock, shares authorized (in shares) 300,000,000 300,000,000
Common stock, shares issued (in shares) 20,848,000 10,143,000
Common stock, shares outstanding (in shares) 20,848,000 10,143,000
v3.25.1
Restatement of Previously Issued Financial Statements - Consolidated Statement of Operations and Comprehensive Loss (Details) - USD ($)
$ / shares in Units, shares in Thousands, $ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Income Statement [Abstract]                        
License revenue $ 1,000     $ 0 $ 0 $ 3,600   $ 3,600 $ 1,000 $ 3,600 $ 1,000 $ 3,600
Operating expenses:                        
Research and development 20,439 $ 17,097 $ 15,410 20,740 20,599 20,869 $ 32,507 41,468 52,946 62,208 77,041 77,486
General and administrative 14,999 15,807 13,789 14,319 13,481 15,586 29,596 29,067 44,595 43,386 63,118 55,056
Total operating expenses 35,438 32,904 29,199 35,059 34,080 36,455 62,103 70,535 97,541 105,594 140,159 132,542
Operating loss (34,438) (32,904) (29,199) (35,059) (34,080) (32,855) (62,103) (66,935) (96,541) (101,994) (139,159) (128,942)
Other income, net 2,087 2,406 2,052 1,661 1,576 1,200 4,458 2,776 6,545 4,437 8,232 5,748
Net loss before income taxes       (33,398) (32,504) (31,655)   (64,159)   (97,557) (130,927) (123,194)
Income tax benefit (provision)       (17) (21) (17)   (38)   (55) 0 1,078
Net loss (32,351) (30,498) (27,147) (33,415) (32,525) (31,672) (57,645) (64,197) (89,996) (97,612) (130,927) (122,116)
Other comprehensive gain:                        
Net unrealized gain on marketable securities 241 (2) (41) 66 198 739 (43) 937 198 1,003 105 1,057
Foreign currency translation adjustment 16 10 (19) (12) (5) (7) (9) (12) 7 (24) (39) 1
Comprehensive loss $ (32,094) $ (30,490) $ (27,207) $ (33,361) $ (32,332) $ (30,940) $ (57,697) $ (63,272) $ (89,791) $ (96,633) $ (130,861) $ (121,058)
Net loss per share - basic (in USD per share) $ (1.55) $ (1.46) $ (1.65) $ (3.31) $ (3.23) $ (3.16) $ (3.08) $ (6.39) $ (4.64) $ (9.69) $ (6.62) $ (12.11)
Net loss per share - diluted (in USD per share) $ (1.55) $ (1.46) $ (1.65) $ (3.31) $ (3.23) $ (3.16) $ (3.08) $ (6.39) $ (4.64) $ (9.69) $ (6.62) $ (12.11)
Weighted-average common shares used to compute net loss per share - basic (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069 19,782 10,082
Weighted-average common shares outstanding - diluted (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069 19,782 10,082
As Previously Reported                        
Income Statement [Abstract]                        
License revenue $ 1,000     $ 0 $ 0 $ 3,600   $ 3,600 $ 1,000 $ 3,600   $ 3,600
Operating expenses:                        
Research and development 20,439 $ 17,097 $ 15,410 20,740 20,599 21,059 $ 32,507 41,658 52,946 62,398   77,676
General and administrative 9,782 3,785 11,429 13,789 12,466 12,780 15,214 25,246 24,996 39,035   49,915
Total operating expenses 30,221 20,882 26,839 34,529 33,065 33,839 47,721 66,904 77,942 101,433   127,591
Operating loss (29,221) (20,882) (26,839) (34,529) (33,065) (30,239) (47,721) (63,304) (76,942) (97,833)   (123,991)
Other income, net 2,087 2,406 2,052 1,661 1,576 1,200 4,458 2,776 6,545 4,437   5,748
Net loss before income taxes       (32,868) (31,489) (29,039)   (60,528)   (93,396)   (118,243)
Income tax benefit (provision)       (17) (21) (17)   (38)   (55)   1,078
Net loss (27,134) (18,476) (24,787) (32,885) (31,510) (29,056) (43,263) (60,566) (70,397) (93,451)   (117,165)
Other comprehensive gain:                        
Net unrealized gain on marketable securities 241 (2) (41) 66 198 739 (43) 937 198 1,003   1,057
Foreign currency translation adjustment 16 10 (19) (12) (5) (7) (9) (12) 7 (24)   1
Comprehensive loss $ (26,877) $ (18,468) $ (24,847) $ (32,831) $ (31,317) $ (28,324) $ (43,315) $ (59,641) $ (70,192) $ (92,472)   $ (116,107)
Net loss per share - basic (in USD per share) $ (1.30) $ (0.89) $ (1.50) $ (3.26) $ (3.13) $ (2.90) $ (2.31) $ (6.02) $ (3.63) $ (9.28)   $ (11.62)
Net loss per share - diluted (in USD per share) $ (1.30) $ (0.89) $ (1.50) $ (3.26) $ (3.13) $ (2.90) $ (2.31) $ (6.02) $ (3.63) $ (9.28)   $ (11.62)
Weighted-average common shares used to compute net loss per share - basic (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069   10,082
Weighted-average common shares outstanding - diluted (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069   10,082
Adjustment                        
Income Statement [Abstract]                        
License revenue $ 0     $ 0 $ 0 $ 0   $ 0 $ 0 $ 0   $ 0
Operating expenses:                        
Research and development 0 $ 0 $ 0 0 0 (190) $ 0 (190) 0 (190)   (190)
General and administrative 5,217 12,022 2,360 530 1,015 2,806 14,382 3,821 19,599 4,351   5,141
Total operating expenses 5,217 12,022 2,360 530 1,015 2,616 14,382 3,631 19,599 4,161   4,951
Operating loss (5,217) (12,022) (2,360) (530) (1,015) (2,616) (14,382) (3,631) (19,599) (4,161)   (4,951)
Other income, net 0 0 0 0 0 0 0 0 0 0   0
Net loss before income taxes       (530) (1,015) (2,616)   (3,631)   (4,161)   (4,951)
Income tax benefit (provision)       0       0
Net loss (5,217) (12,022) (2,360) (530) (1,015) (2,616) (14,382) (3,631) (19,599) (4,161)   (4,951)
Other comprehensive gain:                        
Net unrealized gain on marketable securities 0 0 0 0 0 0 0 0 0 0   0
Foreign currency translation adjustment 0 0 0 0 0 0 0 0 0 0   0
Comprehensive loss $ (5,217) $ (12,022) $ (2,360) $ (530) $ (1,015) $ (2,616) $ (14,382) $ (3,631) $ (19,599) $ (4,161)   $ (4,951)
Net loss per share - basic (in USD per share) $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0   $ 0
Net loss per share - diluted (in USD per share) $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0   $ 0
Weighted-average common shares used to compute net loss per share - basic (in shares) 0 0 0 0 0 0 0 0 0 0   0
Weighted-average common shares outstanding - diluted (in shares) 0 0 0 0 0 0 0 0 0 0   0
v3.25.1
Restatement of Previously Issued Financial Statements - Consolidated Statement of Cash Flows (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Cash flows from operating activities:                        
Net loss $ (32,351) $ (30,498) $ (27,147) $ (33,415) $ (32,525) $ (31,672) $ (57,645) $ (64,197) $ (89,996) $ (97,612) $ (130,927) $ (122,116)
Adjustments to reconcile net loss to net cash used in operating activities:                        
Non-cash lease expense     1,820     2,666 3,683 9,538 5,577 16,382 7,495 18,257
Non-cash sublease income     1,046     (1,567) 4,765 1,219 8,477 435 14,000 (89)
Changes in operating assets and liabilities:                        
Accrued expenses and other liabilities           (939)   (715)   (1,642) 3,021 (5,396)
Supplemental schedule of noncash information                        
Remeasurement of operating lease right-of-use assets and liabilities                     $ 0 7,632
As Previously Reported                        
Cash flows from operating activities:                        
Net loss (27,134) (18,476) (24,787) (32,885) (31,510) (29,056) (43,263) (60,566) (70,397) (93,451)   (117,165)
Adjustments to reconcile net loss to net cash used in operating activities:                        
Non-cash lease expense     506     1,427 1,029 6,936 1,571 12,466   13,027
Non-cash sublease income     0     0 0 0 0 0   0
Changes in operating assets and liabilities:                        
Accrued expenses and other liabilities           (749)   (525)   (1,452)   (5,206)
Supplemental schedule of noncash information                        
Remeasurement of operating lease right-of-use assets and liabilities                       13,711
Adjustment                        
Cash flows from operating activities:                        
Net loss $ (5,217) $ (12,022) (2,360) $ (530) $ (1,015) (2,616) (14,382) (3,631) (19,599) (4,161)   (4,951)
Adjustments to reconcile net loss to net cash used in operating activities:                        
Non-cash lease expense     1,314     1,239 2,654 2,602 4,006 3,916   5,230
Non-cash sublease income     $ 1,046     (1,567) $ 4,765 1,219 $ 8,477 435   (89)
Changes in operating assets and liabilities:                        
Accrued expenses and other liabilities           $ (190)   $ (190)   $ (190)   (190)
Supplemental schedule of noncash information                        
Remeasurement of operating lease right-of-use assets and liabilities                       $ (6,079)
v3.25.1
Summary of Significant Accounting Policies - Additional Information (Details)
12 Months Ended
Dec. 31, 2024
segment
Summary Of Significant Accounting Policies [Line Items]  
Reverse stock split ratio 0.10
Number of reportable segments 1
Number of operating segments 1
Minimum  
Summary Of Significant Accounting Policies [Line Items]  
Estimated useful life of assets (in years) 3 years
Maximum  
Summary Of Significant Accounting Policies [Line Items]  
Estimated useful life of assets (in years) 5 years
v3.25.1
Fair Value Measurements and Fair Value of Financial Instruments - Schedule of Cash Equivalents and Short-term Investments (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]    
Amortized Cost Basis $ 64,974 $ 21,541
Unrealized Gains 66 4
Unrealized Losses (1) (19)
Estimated Fair Value 65,039 21,526
Less: Cash equivalents, amortized cost basis (54,901) (70,972)
Less: Cash equivalents 0 0
Less: Cash equivalents, unrealized losses 8 33
Less: Cash equivalents, estimated fair value (54,893) (70,939)
Total cash equivalents and short-term investments    
Debt Securities, Available-for-sale [Line Items]    
Amortized Cost Basis 119,875 92,513
Unrealized Gains 66 4
Unrealized Losses (9) (52)
Estimated Fair Value 119,932 92,465
Level 1 | Money market funds    
Debt Securities, Available-for-sale [Line Items]    
Amortized Cost Basis 165 10,204
Unrealized Gains 0 0
Unrealized Losses 0 0
Estimated Fair Value 165 10,204
Level 2 | Commercial paper    
Debt Securities, Available-for-sale [Line Items]    
Amortized Cost Basis 78,500 64,693
Unrealized Gains 10 0
Unrealized Losses (9) (35)
Estimated Fair Value 78,501 64,658
Level 2 | U.S. government and agency securities    
Debt Securities, Available-for-sale [Line Items]    
Amortized Cost Basis 41,210 17,616
Unrealized Gains 56 4
Unrealized Losses 0 (17)
Estimated Fair Value $ 41,266 $ 17,603
v3.25.1
Fair Value Measurements and Fair Value of Financial Instruments - Additional Information (Details)
$ in Millions
12 Months Ended
Dec. 31, 2024
USD ($)
security
Dec. 31, 2023
USD ($)
Fair Value Disclosures [Abstract]    
Number of securities, unrealized loss position | security 14  
Marketable securities in an unrealized loss $ 54.2  
Allowance for credit losses 0.0 $ 0.0
Impairment charges on available-for-sale securities $ 0.0 $ 0.0
v3.25.1
Revenue (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Jan. 25, 2021
Sep. 30, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Disaggregation of Revenue [Line Items]                    
License revenue   $ 1,000 $ 0 $ 0 $ 3,600 $ 3,600 $ 1,000 $ 3,600 $ 1,000 $ 3,600
Ray Therapeutics, Inc                    
Disaggregation of Revenue [Line Items]                    
License revenue                 $ 1,000  
Lexeo Therapeutics, Inc                    
Disaggregation of Revenue [Line Items]                    
License fees received $ 7,500                  
v3.25.1
Leases - Additional Information (Details)
$ in Thousands
1 Months Ended 3 Months Ended 12 Months Ended
Jan. 08, 2021
segment
Apr. 07, 2025
USD ($)
Mar. 31, 2025
USD ($)
Feb. 28, 2025
USD ($)
Mar. 31, 2023
USD ($)
Jun. 30, 2023
USD ($)
Dec. 31, 2024
USD ($)
Dec. 31, 2023
USD ($)
Sep. 30, 2024
USD ($)
Jun. 30, 2024
USD ($)
Mar. 31, 2024
USD ($)
Sep. 30, 2023
USD ($)
Sep. 29, 2023
lease
Oct. 26, 2021
USD ($)
Lessee, Lease, Description [Line Items]                            
Restricted cash         $ 2,503 $ 2,650 $ 1,976 $ 1,976 $ 1,976 $ 1,976 $ 1,976 $ 2,650    
Number of facility leases | lease                         2  
Decrease in lease liability         7,900   2,418 3,222            
Non-cash settlement of operating lease liability         $ 14,900   0 14,903            
Deferred rent receivable                 $ 0 $ 0        
Rent expense             13,300 25,300            
Variable lease costs             2,300 2,400            
Operating lease payments             5,800 7,500            
Sublease Tenant | Landlord                            
Lessee, Lease, Description [Line Items]                            
Payments for tenant improvement allowance             20,500 5,400            
Landlord | Sublease Tenant                            
Lessee, Lease, Description [Line Items]                            
Operating lease payments             6,500 $ 5,500            
Building                            
Lessee, Lease, Description [Line Items]                            
Restricted cash             $ 2,000              
Building | CALIFORNIA                            
Lessee, Lease, Description [Line Items]                            
Lease renewal term (in years)             8 years              
Manufacturing Facility                            
Lessee, Lease, Description [Line Items]                            
Weighted-average remaining lease term (in years)             11 years 2 months 12 days 11 years 7 months 6 days            
Weighted-average IBR (as percent)             11.60% 11.70%            
Manufacturing Facility | NORTH CAROLINA                            
Lessee, Lease, Description [Line Items]                            
Lease renewal term (in years) 5 years                          
Number of renewal contracts | segment 2                          
Leasehold improvement allowance                           $ 22,700
Re-measurement of operating lease liabilities           300                
Re-measurement of operating lease right-of-use assets           $ 300                
Deferred rent receivable               $ 0            
Sublease Income             $ 14,000 $ 100            
Manufacturing Facility | NORTH CAROLINA | Subsequent Event | Loss Contingency, Lien On Subtenant's Leasehold Interest                            
Lessee, Lease, Description [Line Items]                            
Amount of lien filed against subtenant       $ 4,800                    
Payments for rent   $ 1,900 $ 1,900 1,400                    
Tenant Improvements       $ 2,300                    
v3.25.1
Leases - Schedule of Future Non-cancellable Lease Payments under Operating Lease (Details)
$ in Thousands
Dec. 31, 2024
USD ($)
Operating Leases  
2025 $ 12,142
2026 14,892
2027 15,278
2028 15,677
2029 16,089
Thereafter 93,087
Total undiscounted lease payments 167,165
Less: Imputed Interest (75,460)
Total $ 91,705
v3.25.1
Balance Sheet Components - Schedule of Property and Equipment, Net (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Dec. 31, 2023
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Property, Plant and Equipment [Line Items]                
Total property and equipment $ 29,652       $ 29,276      
Less accumulated depreciation and amortization (18,045)       (14,512)      
Property and equipment, net 11,607 $ 12,215 $ 13,047 $ 13,766 14,764 $ 15,497 $ 32,041 $ 33,347
Laboratory equipment                
Property, Plant and Equipment [Line Items]                
Total property and equipment 14,963       14,638      
Leasehold improvements                
Property, Plant and Equipment [Line Items]                
Total property and equipment 13,779       13,586      
Computer equipment and software                
Property, Plant and Equipment [Line Items]                
Total property and equipment 901       868      
Construction in progress                
Property, Plant and Equipment [Line Items]                
Total property and equipment $ 9       $ 184      
v3.25.1
Balance Sheet Components - Schedule of Accrued Expenses and Other Current Liabilities (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Dec. 31, 2023
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Balance Sheet Related Disclosures [Abstract]                
Employee compensation $ 9,540       $ 8,040      
Accrued nonclinical, clinical and process development costs 4,401       3,367      
Accrued professional fees 554       351      
State income tax payable 123       101      
Other 1,002       725      
Total accrued expenses and other current liabilities $ 15,620 $ 14,886 $ 9,912 $ 9,160 $ 12,584 $ 16,362 $ 17,704 $ 17,344
v3.25.1
Commitments and Contingencies (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Other Commitments [Line Items]                  
License revenue $ 1,000 $ 0 $ 0 $ 3,600 $ 3,600 $ 1,000 $ 3,600 $ 1,000 $ 3,600
Ray Therapeutics, Inc                  
Other Commitments [Line Items]                  
License revenue               $ 1,000  
Lexeo Therapeutics, Inc                  
Other Commitments [Line Items]                  
License revenue                 $ 3,500
v3.25.1
Stockholders' Equity - Additional Information (Details)
$ / shares in Units, $ in Millions
12 Months Ended
Feb. 07, 2024
USD ($)
director
$ / shares
shares
Dec. 31, 2024
USD ($)
$ / shares
shares
Dec. 31, 2023
USD ($)
$ / shares
shares
Jun. 30, 2024
shares
Oct. 31, 2017
shares
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Accumulated currency translation adjustments | $   $ 0.5 $ 0.4    
Total intrinsic value of stock options exercised | $   $ 0.0 $ 0.0    
Weighted-average fair values of options granted (in USD per share) | $ / shares   $ 8.12 $ 7.96    
Unrecognized stock-based compensation expense related to awards | $   $ 17.1      
Unrecognized stock-based compensation, weighted-average period (in years)   2 years 7 months 6 days      
Stock-Based Compensation          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Shares available for future grants (in shares)   1,277,999      
Options          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Award price as a percentage of grant date fair value, minimum   100.00%      
Incentive Stock Options (ISOs) and Non-Qualified Stock Options (NSOs)          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Award price as a percentage of grant date fair value, minimum   110.00%      
Significant stockholder threshold ownership percentage   10.00%      
Restricted stock units          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Awards granted to employees and non-employees vesting period (in years)   3 years      
Unrecognized stock-based compensation, weighted-average period (in years)   1 year 9 months 18 days      
Total fair values of RSUs vested | $   $ 0.8 $ 1.5    
Unrecognized compensation cost | $   $ 1.7      
2014 Equity Incentive Award Plan          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Percentage of annual increase in number of shares available for future issuance   4.00%      
Shares available for future grants (in shares)       0  
2017 Inducement Plan | Stock Options and Restricted Stock Units          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Common stock shares available for future grant (in shares)         600,000
2014 Plan And Inducement Plan          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Awards granted to employees and non-employees vesting period (in years)   4 years      
2014 Plan And Inducement Plan | Maximum          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Stock options granted period   10 years      
2014 Employee Stock Purchase Plan          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Shares available for future grants (in shares)   533,978      
Common stock shares available for future grant (in shares)   208,833      
Unrecognized compensation cost | $   $ 0.5      
Percentage increase in shares issued   1.00%      
Common stock issued under employee stock purchase plan (in shares)   88,199 82,422    
2024 Equity Incentive Award Plan          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Common stock shares authorized for issuance (in shares)       4,688,345  
Private Placement          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Number of shares issued in transaction (in shares) 10,500,000        
Share price (in USD per share) | $ / shares $ 12.00        
Total gross proceeds | $ $ 127.8        
Private Placement | Two Directors          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Number of shares issued in transaction (in shares) 23,000        
Share price (in USD per share) | $ / shares $ 13.50        
Number of directors | director 2        
Private Placement | Pre-Funded Warrants          
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]          
Common stock issued upon exercise of warrants (in shares) 75,000        
Share price (in USD per share) | $ / shares $ 11.999        
Exercise Price Per Share (in USD per share) | $ / shares $ 0.001        
Beneficial ownership interest limitation (percent)   9.99%      
Warrants exercised (in shares)   0      
v3.25.1
Stockholders' Equity - Schedule of Stock Options Activity (Details) - USD ($)
$ / shares in Units, shares in Thousands, $ in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Options Outstanding    
Beginning Balance (in shares) 2,303  
Granted (in shares) 1,971  
Exercised (in shares) (15)  
Cancelled/forfeited (in shares) (350)  
Ending Balance (in shares) 3,909 2,303
Vested and expected to vest (in shares) 3,909  
Exercisable (in shares) 1,504  
Weighted- Average Exercise Price    
Beginning Balance (in USD per share) $ 48.70  
Granted (in USD per share) 10.39  
Exercised (in USD per share) 9.97  
Cancelled/forfeited (in USD per share) 50.28  
Ending Balance (in USD per share) 29.39 $ 48.70
Vested and expected to vest (in USD per share) 29.39  
Exercisable (in USD per share) $ 58.95  
Weighted- Average Remaining Contract Life (in years)    
Weighted- Average Remaining Contract Life (in years) 8 years 1 month 6 days 6 years 9 months 18 days
Vested and expected to vest 8 years 1 month 6 days  
Exercisable 6 years 6 months  
Outstanding, Aggregate Intrinsic Value $ 0 $ 99
Vested and expected to vest, Aggregate Intrinsic Value 0  
Exercisable, Aggregate Intrinsic Value $ 0  
v3.25.1
Stockholders' Equity - Schedule of Fair Value of Option Issued Valuation Assumptions (Details)
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Options    
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]    
Expected volatility 95.00% 90.00%
Expected term (in years) 6 years 6 years
Expected dividend yield 0.00% 0.00%
Risk-free interest rate 3.90% 4.20%
Employee Stock Purchase Plan    
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]    
Expected volatility 83.00% 78.00%
Expected term (in years) 1 year 3 months 18 days 1 year 3 months 18 days
Expected dividend yield 0.00% 0.00%
Risk-free interest rate 4.30% 5.00%
v3.25.1
Stockholders' Equity - Schedule of Restricted Stock Units Activity (Details) - Restricted stock units - $ / shares
shares in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Number of Units    
Beginning Balance (in shares) 154  
Granted (in shares) 160  
Vested and released (in shares) (29)  
Forfeited (in shares) (26)  
Ending Balance (in shares) 259 154
Weighted- Average Grant Date Fair Value    
Beginning Balance (in USD per share) $ 21.73  
Granted (in USD per share) 12.04  
Vested and released (in USD per share) 26.20  
Forfeited (in USD per share) 14.22  
Ending Balance (in USD per share) $ 16.00 $ 21.73
Weighted- Average Remaining Contractual Term (in years)    
Weighted- Average Remaining Contractual Term (in years) 1 year 2 months 12 days 1 year 4 months 24 days
v3.25.1
Stockholders' Equity - Schedule of Stock-based Compensation Expense (Details) - USD ($)
$ in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]    
Total share-based compensation expense $ 14,350 $ 17,569
Research and development    
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]    
Total share-based compensation expense 4,544 4,969
General and administrative    
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]    
Total share-based compensation expense $ 9,806 $ 12,600
v3.25.1
401(k) Savings Plan (Details) - USD ($)
$ in Millions
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Retirement Benefits [Abstract]    
Contribution by company $ 0.9 $ 0.9
v3.25.1
Segment Reporting (Details)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
USD ($)
Jun. 30, 2024
USD ($)
Mar. 31, 2024
USD ($)
Sep. 30, 2023
USD ($)
Jun. 30, 2023
USD ($)
Mar. 31, 2023
USD ($)
Jun. 30, 2024
USD ($)
Jun. 30, 2023
USD ($)
Sep. 30, 2024
USD ($)
Sep. 30, 2023
USD ($)
Dec. 31, 2024
USD ($)
segment
Dec. 31, 2023
USD ($)
Segment Reporting Information [Line Items]                        
Number of reportable segments | segment                     1  
License revenue $ 1,000     $ 0 $ 0 $ 3,600   $ 3,600 $ 1,000 $ 3,600 $ 1,000 $ 3,600
Less:                        
Total operating expenses 35,438 $ 32,904 $ 29,199 35,059 34,080 36,455 $ 62,103 70,535 97,541 105,594 140,159 132,542
Operating loss (34,438) (32,904) (29,199) (35,059) (34,080) (32,855) (62,103) (66,935) (96,541) (101,994) (139,159) (128,942)
Other income, net (2,087) (2,406) (2,052) (1,661) (1,576) (1,200) (4,458) (2,776) (6,545) (4,437) (8,232) (5,748)
Net loss before income taxes       (33,398) (32,504) (31,655)   (64,159)   (97,557) (130,927) (123,194)
Income tax benefit (provision)       (17) (21) (17)   (38)   (55) 0 1,078
Net loss $ (32,351) $ (30,498) $ (27,147) $ (33,415) $ (32,525) $ (31,672) $ (57,645) $ (64,197) $ (89,996) $ (97,612) (130,927) (122,116)
Reportable Segment                        
Segment Reporting Information [Line Items]                        
License revenue                     1,000 3,600
Less:                        
Compensation and benefits expenses                     56,546 54,572
Clinical trial and manufacturing                     22,333 20,235
Facilities and IT                     37,841 39,114
Other research and development expense                     4,728 4,985
Other segment expenses                     18,711 13,636
Total operating expenses                     140,159 132,542
Operating loss                     (139,159) (128,942)
Other income, net                     (8,232) (5,748)
Net loss before income taxes                     (130,927) (123,194)
Income tax benefit (provision)                     0 1,078
Net loss                     $ (130,927) $ (122,116)
v3.25.1
Income Taxes - Schedule of Components of Loss Before Income Taxes (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2023
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Income Tax Disclosure [Abstract]              
U.S.           $ (130,768) $ (123,040)
Foreign           (159) (154)
Net loss before income taxes $ (33,398) $ (32,504) $ (31,655) $ (64,159) $ (97,557) $ (130,927) $ (123,194)
v3.25.1
Income Taxes- Schedule of Components of Income Tax (Benefit) Provision (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2023
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Current:              
Foreign           $ 0 $ (1,078)
Total current tax (benefit) provision           0 (1,078)
Deferred              
Foreign           0 0
Total deferred tax provision           0 0
Total income tax (benefit) provision $ 17 $ 21 $ 17 $ 38 $ 55 $ 0 $ (1,078)
v3.25.1
Income Taxes - Schedule of Reconciliation of Income Tax Expense Computed Statutory Federal Income Tax and Financial Statements (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2023
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Income Tax Disclosure [Abstract]              
Federal income tax benefit at statutory rate           $ (27,495) $ (25,871)
Stock compensation           3,189 6,001
Non-deductible expenses           41 21
Research and development tax credits           (5,642) (1,810)
Change in valuation allowance           23,801 19,558
Foreign rate differential           (14) (14)
Impact of internal reorganization           0 1,323
Uncertain tax positions           6,100 (1,078)
Other           20 792
Total income tax (benefit) provision $ 17 $ 21 $ 17 $ 38 $ 55 $ 0 $ (1,078)
v3.25.1
Income Taxes - Schedule of Deferred Tax Assets (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Dec. 31, 2023
Deferred tax assets:    
Net operating loss carryforwards $ 137,140 $ 128,171
Accruals, reserve and other 1,972 1,749
Tax credit carryforwards 29,263 21,254
Stock-based compensation 6,349 7,274
Intangibles 7 11
Lease obligation 19,622 17,322
Capital losses 9,850 9,850
Section 174 R&D capitalization 35,842 29,688
Other 5 3
Total deferred tax assets before valuation allowance 240,050 215,322
Valuation allowance (232,058) (205,861)
Total deferred tax assets 7,992 9,461
Deferred tax liabilities:    
Right-of-use assets (7,149) (8,153)
Property and equipment (843) (1,308)
Total deferred tax liabilities (7,992) (9,461)
Net deferred tax assets $ 0 $ 0
v3.25.1
Income Taxes - Additional Information (Details) - USD ($)
$ in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Dec. 31, 2022
Income Taxes [Line Items]      
Research and development tax credits $ (5,642) $ (1,810)  
Increase in valuation allowance due to net operating loss 26,200 17,700  
Unrecognized tax benefits 27,188 22,758 $ 23,069
Accrued interest or penalties related to uncertain tax positions 0 0  
Interest and penalties recognized 0 0  
CALIFORNIA      
Income Taxes [Line Items]      
Research and development tax credits   3,400  
Decrease in unrecognized tax benefits   $ 1,900  
Domestic tax authority      
Income Taxes [Line Items]      
NOL carryforwards 552,700    
NOL carryforwards subject to expiration 57,300    
Domestic tax authority | Research Tax Credit Carryforward      
Income Taxes [Line Items]      
Tax credit carryforwards 25,100    
State credit carryforwards      
Income Taxes [Line Items]      
NOL carryforwards 345,200    
State credit carryforwards | Research Tax Credit Carryforward      
Income Taxes [Line Items]      
Tax credit carryforwards 13,800    
Foreign Tax Jurisdiction      
Income Taxes [Line Items]      
NOL carryforwards $ 49,500    
v3.25.1
Income Taxes - Schedule of Unrecognized Tax Benefits (Details) - USD ($)
$ in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Reconciliation of Unrecognized Tax Benefits [Roll Forward]    
Unrecognized tax benefits as of the beginning of the year $ 22,758 $ 23,069
Increase related to tax positions taken during the prior year 0 43
Decrease related to tax positions taken during the prior year (2,880) 0
Increase related to tax position taken during the current year 7,310 3,121
Decrease related to tax position taken during the current year 0 (3,475)
Unrecognized tax benefits as of the end of the year $ 27,188 $ 22,758
v3.25.1
Net Loss per Share (Details) - shares
shares in Thousands
12 Months Ended
Dec. 31, 2024
Dec. 31, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]    
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share (in shares) 4,200 2,474
Stock options    
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]    
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share (in shares) 3,909 2,303
Restricted stock units    
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]    
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share (in shares) 259 154
ESPP    
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]    
Antidilutive common stock equivalents excluded from calculation of diluted net loss per share (in shares) 32 17
v3.25.1
Quarterly Financial Information (Unaudited) - Additional Information (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Interim Period, Costs Not Allocable [Line Items]                        
Deferred rent receivable $ 0 $ 0         $ 0   $ 0      
Research and development expense reversal (20,439) (17,097) $ (15,410) $ (20,740) $ (20,599) $ (20,869) (32,507) $ (41,468) (52,946) $ (62,208) $ (77,041) $ (77,486)
Right-to-use asset 33,680 33,996 34,478 35,599 41,233 46,598 33,996 41,233 33,680 35,599 33,611 35,024
Lease liability                     $ 91,705  
Adjustment                        
Interim Period, Costs Not Allocable [Line Items]                        
Deferred rent receivable (7,116) (6,963)         (6,963)   (7,116)      
Research and development expense reversal 0 0 0 0 0 190 0 190 0 190   190
Right-to-use asset $ (17,015) $ (17,241) $ (17,282) $ (17,158) $ (17,054) (22,905) $ (17,241) $ (17,054) $ (17,015) $ (17,158)   $ (17,242)
Adjustment | Redwood, California Facility Lease                        
Interim Period, Costs Not Allocable [Line Items]                        
Right-to-use asset           300            
Lease liability           $ 300            
v3.25.1
Quarterly Financial Information (Unaudited) - Schedule Of Quarterly Information - Condensed Consolidated Balance Sheet (Details) - USD ($)
$ in Thousands
Dec. 31, 2024
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Dec. 31, 2023
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Dec. 31, 2022
Current assets:                  
Cash and cash equivalents $ 60,652 $ 92,851 $ 127,321 $ 149,608 $ 75,000 $ 105,366 $ 111,187 $ 67,552  
Short-term investments 65,039 60,390 46,506 43,720 21,526 11,716 30,266 96,733  
Prepaid expenses and other current assets 5,609 7,839 5,591 5,802 6,247 7,514 5,690 4,588  
Total current assets 131,300 161,080 179,418 199,130 102,773 124,596 147,143 168,873  
Operating lease right-of-use assets 33,611 33,680 33,996 34,478 35,024 35,599 41,233 46,598  
Property and equipment, net 11,607 12,215 13,047 13,766 14,764 15,497 32,041 33,347  
Deferred rent receivable   0 0            
Restricted cash 1,976 1,976 1,976 1,976 1,976 2,650 2,650 2,503  
Deposit and other long-term assets 1,347 1,293 1,162 1,196 1,231 1,270 1,308 1,351  
Total assets 179,841 210,244 229,599 250,546 155,768 179,612 224,375 252,672  
Current liabilities:                  
Accounts payable 1,610 2,110 1,973 1,897 1,921 2,170 1,418 2,863  
Accrued expenses and other current liabilities 15,620 14,886 9,912 9,160 12,584 16,362 17,704 17,344  
Lease liability, current portion 5,668 5,617 5,570 5,521 5,476 5,427 20,535 20,734  
Total current liabilities 22,898 22,613 17,455 16,578 19,981 23,959 39,657 40,941  
Long-term liabilities:                  
Lease liability, net of current portion 86,037 79,361 74,744 70,287 68,565 68,396 68,490 67,886  
Total liabilities 109,127 101,974 92,199 86,865 88,546 92,355 108,147 108,827  
Commitments and contingencies  
Stockholders’ equity:                  
Preferred stock 0 0 0 0 0 0 0 0  
Common stock 2 2 2 2 1 1 1 1  
Additional paid-in capital 1,138,070 1,134,556 1,131,592 1,127,383 1,003,718 999,328 994,938 990,223  
Accumulated other comprehensive loss (407) (268) (525) (533) (473) (552) (606) (799)  
Accumulated deficit (1,066,951) (1,026,020) (993,669) (963,171) (936,024) (911,520) (878,105) (845,580)  
Total stockholders’ equity 70,714 108,270 137,400 163,681 67,222 87,257 116,228 143,845 $ 170,222
Total liabilities and stockholders’ equity $ 179,841 210,244 229,599 250,546 155,768 179,612 224,375 252,672  
As Previously Reported                  
Current assets:                  
Cash and cash equivalents   92,851 127,321 149,608 75,000 105,366 111,187 67,552  
Short-term investments   60,390 46,506 43,720 21,526 11,716 30,266 96,733  
Prepaid expenses and other current assets   7,839 5,591 5,802 6,247 7,514 5,690 4,588  
Total current assets   161,080 179,418 199,130 102,773 124,596 147,143 168,873  
Operating lease right-of-use assets   50,695 51,237 51,760 52,266 52,757 58,287 69,503  
Property and equipment, net   12,215 13,047 13,766 14,764 15,497 32,041 33,347  
Deferred rent receivable   7,116 6,963            
Restricted cash   1,976 1,976 1,976 1,976 2,650 2,650 2,503  
Deposit and other long-term assets   1,293 1,162 1,196 1,231 1,270 1,308 1,351  
Total assets   234,375 253,803 267,828 173,010 196,770 241,429 275,577  
Current liabilities:                  
Accounts payable   2,110 1,973 1,897 1,921 2,170 1,418 2,863  
Accrued expenses and other current liabilities   14,886 9,912 9,160 12,584 16,362 17,704 17,344  
Lease liability, current portion   10,661 10,576 10,492 10,409 10,324 25,396 26,265  
Total current liabilities   27,657 22,461 21,549 24,914 28,856 44,518 46,472  
Long-term liabilities:                  
Lease liability, net of current portion   62,602 63,313 63,991 64,627 65,200 65,756 71,348  
Total liabilities   90,259 85,774 85,540 89,541 94,056 110,274 117,820  
Commitments and contingencies    
Stockholders’ equity:                  
Preferred stock   0 0 0 0 0 0 0  
Common stock   2 2 2 1 1 1 1  
Additional paid-in capital   1,134,556 1,131,592 1,127,383 1,003,718 999,328 994,938 990,223  
Accumulated other comprehensive loss   (268) (525) (533) (473) (552) (606) (799)  
Accumulated deficit   (990,174) (963,040) (944,564) (919,777) (896,063) (863,178) (831,668)  
Total stockholders’ equity   144,116 168,029 182,288 83,469 102,714 131,155 157,757  
Total liabilities and stockholders’ equity   234,375 253,803 267,828 173,010 196,770 241,429 275,577  
Adjustment                  
Current assets:                  
Cash and cash equivalents   0 0 0 0 0 0 0  
Short-term investments   0 0 0 0 0 0 0  
Prepaid expenses and other current assets   0 0 0 0 0 0  
Total current assets   0 0 0 0 0 0 0  
Operating lease right-of-use assets   (17,015) (17,241) (17,282) (17,242) (17,158) (17,054) (22,905)  
Property and equipment, net   0 0 0 0 0 0  
Deferred rent receivable   (7,116) (6,963)            
Restricted cash   0 0 0 0 0 0 0  
Deposit and other long-term assets   0 0 0 0 0 0  
Total assets   (24,131) (24,204) (17,282) (17,242) (17,158) (17,054) (22,905)  
Current liabilities:                  
Accounts payable   0 0 0 0 0 0 0  
Accrued expenses and other current liabilities   0 0 0 0 0 0 0  
Lease liability, current portion   (5,044) (5,006) (4,971) (4,933) (4,897) (4,861) (5,531)  
Total current liabilities   (5,044) (5,006) (4,971) (4,933) (4,897) (4,861) (5,531)  
Long-term liabilities:                  
Lease liability, net of current portion   16,759 11,431 6,296 3,938 3,196 2,734 (3,462)  
Total liabilities   11,715 6,425 1,325 (995) (1,701) (2,127) (8,993)  
Commitments and contingencies    
Stockholders’ equity:                  
Preferred stock   0 0 0 0 0 0 0  
Common stock   0 0 0 0 0 0 0  
Additional paid-in capital   0 0 0 0 0 0 0  
Accumulated other comprehensive loss   0 0 0 0 0 0 0  
Accumulated deficit   (35,846) (30,629) (18,607) (16,247) (15,457) (14,927) (13,912) $ (11,300)
Total stockholders’ equity   (35,846) (30,629) (18,607) (16,247) (15,457) (14,927) (13,912)  
Total liabilities and stockholders’ equity   $ (24,131) $ (24,204) $ (17,282) $ (17,242) $ (17,158) $ (17,054) $ (22,905)  
v3.25.1
Quarterly Financial Information (Unaudited) - Schedule Of Quarterly Information - Condensed Consolidated Statements of Operations and Comprehensive Loss (Details) - USD ($)
$ / shares in Units, shares in Thousands, $ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Income Statement [Abstract]                        
License revenue $ 1,000     $ 0 $ 0 $ 3,600   $ 3,600 $ 1,000 $ 3,600 $ 1,000 $ 3,600
Operating expenses:                        
Research and development 20,439 $ 17,097 $ 15,410 20,740 20,599 20,869 $ 32,507 41,468 52,946 62,208 77,041 77,486
General and administrative 14,999 15,807 13,789 14,319 13,481 15,586 29,596 29,067 44,595 43,386 63,118 55,056
Total operating expenses 35,438 32,904 29,199 35,059 34,080 36,455 62,103 70,535 97,541 105,594 140,159 132,542
Operating loss (34,438) (32,904) (29,199) (35,059) (34,080) (32,855) (62,103) (66,935) (96,541) (101,994) (139,159) (128,942)
Other income, net 2,087 2,406 2,052 1,661 1,576 1,200 4,458 2,776 6,545 4,437 8,232 5,748
Net loss before income taxes       (33,398) (32,504) (31,655)   (64,159)   (97,557) (130,927) (123,194)
Income tax benefit (provision)       (17) (21) (17)   (38)   (55) 0 1,078
Net loss (32,351) (30,498) (27,147) (33,415) (32,525) (31,672) (57,645) (64,197) (89,996) (97,612) (130,927) (122,116)
Other comprehensive gain (loss):                        
Net unrealized gain on marketable securities 241 (2) (41) 66 198 739 (43) 937 198 1,003 105 1,057
Foreign currency translation adjustment 16 10 (19) (12) (5) (7) (9) (12) 7 (24) (39) 1
Comprehensive loss $ (32,094) $ (30,490) $ (27,207) $ (33,361) $ (32,332) $ (30,940) $ (57,697) $ (63,272) $ (89,791) $ (96,633) $ (130,861) $ (121,058)
Net loss per share - basic (in USD per share) $ (1.55) $ (1.46) $ (1.65) $ (3.31) $ (3.23) $ (3.16) $ (3.08) $ (6.39) $ (4.64) $ (9.69) $ (6.62) $ (12.11)
Net loss per share - diluted (in USD per share) $ (1.55) $ (1.46) $ (1.65) $ (3.31) $ (3.23) $ (3.16) $ (3.08) $ (6.39) $ (4.64) $ (9.69) $ (6.62) $ (12.11)
Weighted-average common shares used to compute net loss per share - basic (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069 19,782 10,082
Weighted-average common shares outstanding - diluted (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069 19,782 10,082
As Previously Reported                        
Income Statement [Abstract]                        
License revenue $ 1,000     $ 0 $ 0 $ 3,600   $ 3,600 $ 1,000 $ 3,600   $ 3,600
Operating expenses:                        
Research and development 20,439 $ 17,097 $ 15,410 20,740 20,599 21,059 $ 32,507 41,658 52,946 62,398   77,676
General and administrative 9,782 3,785 11,429 13,789 12,466 12,780 15,214 25,246 24,996 39,035   49,915
Total operating expenses 30,221 20,882 26,839 34,529 33,065 33,839 47,721 66,904 77,942 101,433   127,591
Operating loss (29,221) (20,882) (26,839) (34,529) (33,065) (30,239) (47,721) (63,304) (76,942) (97,833)   (123,991)
Other income, net 2,087 2,406 2,052 1,661 1,576 1,200 4,458 2,776 6,545 4,437   5,748
Net loss before income taxes       (32,868) (31,489) (29,039)   (60,528)   (93,396)   (118,243)
Income tax benefit (provision)       (17) (21) (17)   (38)   (55)   1,078
Net loss (27,134) (18,476) (24,787) (32,885) (31,510) (29,056) (43,263) (60,566) (70,397) (93,451)   (117,165)
Other comprehensive gain (loss):                        
Net unrealized gain on marketable securities 241 (2) (41) 66 198 739 (43) 937 198 1,003   1,057
Foreign currency translation adjustment 16 10 (19) (12) (5) (7) (9) (12) 7 (24)   1
Comprehensive loss $ (26,877) $ (18,468) $ (24,847) $ (32,831) $ (31,317) $ (28,324) $ (43,315) $ (59,641) $ (70,192) $ (92,472)   $ (116,107)
Net loss per share - basic (in USD per share) $ (1.30) $ (0.89) $ (1.50) $ (3.26) $ (3.13) $ (2.90) $ (2.31) $ (6.02) $ (3.63) $ (9.28)   $ (11.62)
Net loss per share - diluted (in USD per share) $ (1.30) $ (0.89) $ (1.50) $ (3.26) $ (3.13) $ (2.90) $ (2.31) $ (6.02) $ (3.63) $ (9.28)   $ (11.62)
Weighted-average common shares used to compute net loss per share - basic (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069   10,082
Weighted-average common shares outstanding - diluted (in shares) 20,876 20,852 16,479 10,099 10,076 10,030 18,713 10,053 19,408 10,069   10,082
Adjustment                        
Income Statement [Abstract]                        
License revenue $ 0     $ 0 $ 0 $ 0   $ 0 $ 0 $ 0   $ 0
Operating expenses:                        
Research and development 0 $ 0 $ 0 0 0 (190) $ 0 (190) 0 (190)   (190)
General and administrative 5,217 12,022 2,360 530 1,015 2,806 14,382 3,821 19,599 4,351   5,141
Total operating expenses 5,217 12,022 2,360 530 1,015 2,616 14,382 3,631 19,599 4,161   4,951
Operating loss (5,217) (12,022) (2,360) (530) (1,015) (2,616) (14,382) (3,631) (19,599) (4,161)   (4,951)
Other income, net 0 0 0 0 0 0 0 0 0 0   0
Net loss before income taxes       (530) (1,015) (2,616)   (3,631)   (4,161)   (4,951)
Income tax benefit (provision)       0       0
Net loss (5,217) (12,022) (2,360) (530) (1,015) (2,616) (14,382) (3,631) (19,599) (4,161)   (4,951)
Other comprehensive gain (loss):                        
Net unrealized gain on marketable securities 0 0 0 0 0 0 0 0 0 0   0
Foreign currency translation adjustment 0 0 0 0 0 0 0 0 0 0   0
Comprehensive loss $ (5,217) $ (12,022) $ (2,360) $ (530) $ (1,015) $ (2,616) $ (14,382) $ (3,631) $ (19,599) $ (4,161)   $ (4,951)
Net loss per share - basic (in USD per share) $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0   $ 0
Net loss per share - diluted (in USD per share) $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0 $ 0   $ 0
Weighted-average common shares used to compute net loss per share - basic (in shares) 0 0 0 0 0 0 0 0 0 0   0
Weighted-average common shares outstanding - diluted (in shares) 0 0 0 0 0 0 0 0 0 0   0
v3.25.1
Quarterly Financial Information (Unaudited) - Schedule Of Restatements Of Quarterly Information - Condensed Consolidated Statements of Cash Flows (Details) - USD ($)
$ in Thousands
3 Months Ended 6 Months Ended 9 Months Ended 12 Months Ended
Sep. 30, 2024
Jun. 30, 2024
Mar. 31, 2024
Sep. 30, 2023
Jun. 30, 2023
Mar. 31, 2023
Jun. 30, 2024
Jun. 30, 2023
Sep. 30, 2024
Sep. 30, 2023
Dec. 31, 2024
Dec. 31, 2023
Cash flows from operating activities:                        
Net loss $ (32,351) $ (30,498) $ (27,147) $ (33,415) $ (32,525) $ (31,672) $ (57,645) $ (64,197) $ (89,996) $ (97,612) $ (130,927) $ (122,116)
Adjustments to reconcile net loss to net cash used in operating activities:                        
Non-cash lease expense     1,820     2,666 3,683 9,538 5,577 16,382 7,495 18,257
Non-cash sublease loss/(income)     1,046     (1,567) 4,765 1,219 8,477 435 14,000 (89)
Changes in operating assets and liabilities:                        
Deposit and other long-term assets and deferred rent receivable             69   (62)   (68) 182
Accrued expenses and other liabilities           (939)   (715)   (1,642) $ 3,021 (5,396)
Supplemental schedule of noncash information                        
Remeasurement of operating lease right-of-use assets           7,900   7,632   7,632    
As Previously Reported                        
Cash flows from operating activities:                        
Net loss (27,134) (18,476) (24,787) (32,885) (31,510) (29,056) (43,263) (60,566) (70,397) (93,451)   (117,165)
Adjustments to reconcile net loss to net cash used in operating activities:                        
Non-cash lease expense     506     1,427 1,029 6,936 1,571 12,466   13,027
Non-cash sublease loss/(income)     0     0 0 0 0 0   0
Changes in operating assets and liabilities:                        
Deposit and other long-term assets and deferred rent receivable             (6,894)   (7,178)      
Accrued expenses and other liabilities           (749)   (525)   (1,452)   (5,206)
Supplemental schedule of noncash information                        
Remeasurement of operating lease right-of-use assets           8,004   13,711   13,711    
Adjustment                        
Cash flows from operating activities:                        
Net loss $ (5,217) $ (12,022) (2,360) $ (530) $ (1,015) (2,616) (14,382) (3,631) (19,599) (4,161)   (4,951)
Adjustments to reconcile net loss to net cash used in operating activities:                        
Non-cash lease expense     1,314     1,239 2,654 2,602 4,006 3,916   5,230
Non-cash sublease loss/(income)     $ 1,046     (1,567) 4,765 1,219 8,477 435   (89)
Changes in operating assets and liabilities:                        
Deposit and other long-term assets and deferred rent receivable             $ 6,963   $ 7,116      
Accrued expenses and other liabilities           (190)   (190)   (190)   $ (190)
Supplemental schedule of noncash information                        
Remeasurement of operating lease right-of-use assets           $ (104)   $ (6,079)   $ (6,079)    
v3.25.1
Subsequent Events (Details) - USD ($)
$ in Thousands
1 Months Ended
Apr. 07, 2025
Mar. 31, 2025
Feb. 28, 2025
Dec. 31, 2024
Subsequent Event [Line Items]        
Total undiscounted lease payments       $ 167,165
Remaining lease obligation due between April 8 and December 31, 2025       $ 12,142
Subsequent Event | NORTH CAROLINA | Manufacturing Facility | Loss Contingency, Lien On Subtenant's Leasehold Interest        
Subsequent Event [Line Items]        
Amounts due     $ 4,800  
Payments for rent $ 1,900 $ 1,900 $ 1,400  
Total undiscounted lease payments 119,700      
Remaining lease obligation due between April 8 and December 31, 2025 $ 5,700