Voyager Therapeutics (VYGR) 10-Q 12 Nov 24 2024 Q3 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	9 Months Ended
	Sep. 30, 2024	Oct. 28, 2024
Document and Entity Information
Document Type	10-Q
Document Quarterly Report	true
Document Transition Report	false
Document Period End Date	Sep. 30, 2024
Entity File Number	001-37625
Entity Registrant Name	Voyager Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	46-3003182
Entity Address, Address Line One	75 Hayden Avenue
Entity Address, City or Town	Lexington
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02421
City Area Code	857
Local Phone Number	259-5340
Title of 12(b) Security	Common Stock, $0.001 par value
Trading Symbol	VYGR
Security Exchange Name	NASDAQ
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
Entity Shell Company	false
Entity Central Index Key	0001640266
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q3
Entity Common Stock, Shares Outstanding		54,625,586

Condensed Consolidated Balance

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 112,525	$ 68,802
Marketable securities, current	232,835	162,073
Accounts receivable	15,954	80,150
Related party collaboration receivable	3,714	3,341
Prepaid expenses and other current assets	8,415	5,318
Total current assets	373,443	319,684
Property and equipment, net	15,201	16,494
Deposits and other non-current assets	2,874	1,593
Operating lease, right-of-use assets	34,523	13,510
Total assets	426,041	351,281
Current liabilities:
Accounts payable	3,160	1,604
Accrued expenses	11,442	16,823
Other current liabilities	6,979	3,200
Deferred revenue, current	22,629	42,881
Total current liabilities	44,210	64,508
Deferred revenue, non-current	12,153	32,359
Other non-current liabilities	39,368	18,094
Total liabilities	95,731	114,961
Commitments and contingencies (see note 7)
Stockholders' equity:
Preferred stock, $0.001 par value: 5,000,000 shares authorized at September 30, 2024 and December 31, 2023; no shares issued and outstanding at September 30, 2024 and December 31, 2023
Common stock, $0.001 par value: 120,000,000 shares authorized at September 30, 2024 and December 31, 2023; 54,542,048 and 44,038,333 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively	55	44
Additional paid-in capital	621,985	497,506
Accumulated other comprehensive loss	(33)	(48)
Accumulated deficit	(291,697)	(261,182)
Total stockholders' equity	330,310	236,320
Total liabilities and stockholders' equity	$ 426,041	$ 351,281

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 2024	Dec. 31, 2023
Condensed Consolidated Balance Sheets
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized	5,000,000	5,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock shares authorized	120,000,000	120,000,000
Common stock shares issued	54,542,048	44,038,333
Common stock share outstanding	54,542,048	44,038,333

Condensed Consolidated Statemen

Condensed Consolidated Statements of Operations and Comprehensive (Loss) Income - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Condensed Consolidated Statements of Operations and Comprehensive (Loss) Income
Collaboration revenue	$ 24,629	$ 4,614	$ 73,723	$ 159,947
Operating expenses:
Research and development	30,241	25,863	91,785	66,416
General and administrative	8,168	8,258	26,926	25,580
Total operating expenses	38,409	34,121	118,711	91,996
Operating (loss) income	(13,780)	(29,507)	(44,988)	67,951
Other income:
Interest income	4,579	3,429	14,334	8,567
Other income	200		220	3
Total other income, net	4,779	3,429	14,554	8,570
(Loss) income before income taxes	(9,001)	(26,078)	(30,434)	76,521
Income tax provision (benefit)	43	(177)	81	586
Net (loss) income	(9,044)	(25,901)	(30,515)	75,935
Other comprehensive (loss) income:
Net unrealized gain (loss) on available-for-sale securities	489	(115)	15	(29)
Total other comprehensive income (loss)	489	(115)	15	(29)
Comprehensive (loss) income	$ (8,555)	$ (26,016)	$ (30,500)	$ 75,906
Net (loss) income per share, basic	$ (0.16)	$ (0.59)	$ (0.53)	$ 1.85
Net (loss) income per share, diluted	$ (0.16)	$ (0.59)	$ (0.53)	$ 1.78
Weighted-average common shares outstanding, basic	57,851,110	43,864,838	57,564,413	40,962,116
Weighted-average common shares outstanding, diluted	57,851,110	43,864,838	57,564,413	42,610,724

Condensed Consolidated Statem_2

Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Common stock 2023 Neurocrine Collaborative Agreement	Common stock 2023 Novartis Collaboration Agreement	Common stock	Additional paid-in capital 2023 Neurocrine Collaborative Agreement	Additional paid-in capital 2023 Novartis Collaboration Agreement	Additional paid-in capital	Accumulated other comprehensive loss	Accumulated deficit	2023 Neurocrine Collaborative Agreement	2023 Novartis Collaboration Agreement	Total
Stockholders' Equity, Beginning Balance at Dec. 31, 2022			$ 38			$ 452,713	$ (219)	$ (393,512)			$ 59,020
Shares, Outstanding, Beginning Balance at Dec. 31, 2022			38,613,891
Exercises of vested stock options						185					185
Exercises of vested stock options (in shares)			51,993
Vesting of restricted stock units (in shares)			374,417
Issuance of common stock	$ 5			$ 31,116					$ 31,121
Issuance of common stock (in shares)	4,395,588
Stock based compensation expense						2,504					2,504
Unrealized gain (loss) on available-for-sale securities, net of tax							87				87
Net income (loss)								124,044			124,044
Stockholders' Equity, Ending Balance at Mar. 31, 2023			$ 43			486,518	(132)	(269,468)			216,961
Shares, Outstanding, Ending Balance at Mar. 31, 2023			43,435,889
Stockholders' Equity, Beginning Balance at Dec. 31, 2022			$ 38			452,713	(219)	(393,512)			59,020
Shares, Outstanding, Beginning Balance at Dec. 31, 2022			38,613,891
Unrealized gain (loss) on available-for-sale securities, net of tax											(29)
Net income (loss)											75,935
Stockholders' Equity, Ending Balance at Sep. 30, 2023			$ 44			494,001	(248)	(317,577)			176,220
Shares, Outstanding, Ending Balance at Sep. 30, 2023			43,909,161
Stockholders' Equity, Beginning Balance at Mar. 31, 2023			$ 43			486,518	(132)	(269,468)			216,961
Shares, Outstanding, Beginning Balance at Mar. 31, 2023			43,435,889
Exercises of vested stock options			$ 1			1,228					1,229
Exercises of vested stock options (in shares)			198,348
Vesting of restricted stock units (in shares)			62,828
Issuance of common stock under ESPP						418					418
Issuance of common stock under ESPP (in shares)			62,344
Stock based compensation expense						2,627					2,627
Unrealized gain (loss) on available-for-sale securities, net of tax							(1)				(1)
Net income (loss)								(22,208)			(22,208)
Stockholders' Equity, Ending Balance at Jun. 30, 2023			$ 44			490,791	(133)	(291,676)			199,026
Shares, Outstanding, Ending Balance at Jun. 30, 2023			43,759,409
Exercises of vested stock options						415					415
Exercises of vested stock options (in shares)			127,252
Vesting of restricted stock units (in shares)			22,500
Stock based compensation expense						2,795					2,795
Unrealized gain (loss) on available-for-sale securities, net of tax							(115)				(115)
Net income (loss)								(25,901)			(25,901)
Stockholders' Equity, Ending Balance at Sep. 30, 2023			$ 44			494,001	(248)	(317,577)			176,220
Shares, Outstanding, Ending Balance at Sep. 30, 2023			43,909,161
Stockholders' Equity, Beginning Balance at Dec. 31, 2023			$ 44			497,506	(48)	(261,182)			236,320
Shares, Outstanding, Beginning Balance at Dec. 31, 2023			44,038,333
Exercises of vested stock options						78					78
Exercises of vested stock options (in shares)			32,500
Vesting of restricted stock units (in shares)			324,520
Issuance of common stock		$ 2			$ 19,303					$ 19,305
Issuance of common stock (in shares)		2,145,002
Issuance of common stock and pre-funded warrants in connection with underwritten public offering			$ 8			93,465					93,473
Issuance of common stock and pre-funded warrants in connection with underwritten public offering (in shares )			7,777,778
Stock based compensation expense						3,498					3,498
Unrealized gain (loss) on available-for-sale securities, net of tax							(458)				(458)
Net income (loss)								(11,330)			(11,330)
Stockholders' Equity, Ending Balance at Mar. 31, 2024			$ 54			613,850	(506)	(272,512)			340,886
Shares, Outstanding, Ending Balance at Mar. 31, 2024			54,318,133
Stockholders' Equity, Beginning Balance at Dec. 31, 2023			$ 44			497,506	(48)	(261,182)			$ 236,320
Shares, Outstanding, Beginning Balance at Dec. 31, 2023			44,038,333
Exercises of vested stock options (in shares)											86,100
Unrealized gain (loss) on available-for-sale securities, net of tax											$ 15
Net income (loss)											(30,515)
Stockholders' Equity, Ending Balance at Sep. 30, 2024			$ 55			621,985	(33)	(291,697)			330,310
Shares, Outstanding, Ending Balance at Sep. 30, 2024			54,542,048
Stockholders' Equity, Beginning Balance at Mar. 31, 2024			$ 54			613,850	(506)	(272,512)			340,886
Shares, Outstanding, Beginning Balance at Mar. 31, 2024			54,318,133
Exercises of vested stock options						90					90
Exercises of vested stock options (in shares)			25,958
Vesting of restricted stock units (in shares)			56,549
Issuance of common stock under ESPP						639					639
Issuance of common stock under ESPP (in shares)			71,473
Stock based compensation expense						3,959					3,959
Unrealized gain (loss) on available-for-sale securities, net of tax							(16)				(16)
Net income (loss)								(10,141)			(10,141)
Stockholders' Equity, Ending Balance at Jun. 30, 2024			$ 54			618,538	(522)	(282,653)			335,417
Shares, Outstanding, Ending Balance at Jun. 30, 2024			54,472,113
Exercises of vested stock options						92					92
Exercises of vested stock options (in shares)			27,642
Vesting of restricted stock units			$ 1								1
Vesting of restricted stock units (in shares)			42,293
Stock based compensation expense						3,355					3,355
Unrealized gain (loss) on available-for-sale securities, net of tax							489				489
Net income (loss)								(9,044)			(9,044)
Stockholders' Equity, Ending Balance at Sep. 30, 2024			$ 55			$ 621,985	$ (33)	$ (291,697)			$ 330,310
Shares, Outstanding, Ending Balance at Sep. 30, 2024			54,542,048

Condensed Consolidated Statem_3

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Cash flow from operating activities
Net (loss) income	$ (30,515)	$ 75,935
Adjustments to reconcile net (loss) income to net cash (used in) provided by operating activities:
Stock-based compensation expense	11,048	8,108
Depreciation	3,720	3,106
Impairment charge on leased facility	2,776
Amortization of premiums and discounts on marketable securities	(6,456)	(1,832)
Loss on disposal of fixed assets	478	143
Changes in operating assets and liabilities:
Accounts receivable	64,196
Related party collaboration receivable	(373)	(2,996)
Prepaid expenses and other current assets	(3,097)	(342)
Operating lease, right-of-use asset	3,423	1,459
Accounts payable	1,556	704
Accrued expenses	(5,381)	3,236
Operating lease liabilities	(1,698)	(2,102)
Deferred revenue	(40,458)	16,260
Net cash (used in) provided by operating activities	(781)	101,679
Cash flow from investing activities
Purchases of property and equipment	(3,366)	(2,501)
Purchases of marketable securities	(334,667)	(194,975)
Proceeds from sales and maturities of marketable securities	270,377	29,000
Net cash used in investing activities	(67,656)	(168,476)
Cash flow from financing activities
Proceeds from the exercise of stock options	261	1,829
Proceeds from the issuance of common stock in connection with the underwritten public offering	93,473
Proceeds from the issuance of common stock in connection with the 2023 Novartis Stock Purchase Agreement	19,305
Proceeds from the issuance of common stock in connection with the 2023 Neurocrine Collaboration Agreement		31,121
Proceeds from the purchase of common stock under ESPP	402	235
Net cash provided by financing activities	113,441	33,185
Net increase (decrease) in cash, cash equivalents, and restricted cash	45,004	(33,612)
Cash, cash equivalents, and restricted cash, beginning of period	70,395	100,474
Cash, cash equivalents, and restricted cash, end of period	115,399	$ 66,862
Supplemental disclosure of cash and non-cash activities
Operating lease right-of-use asset obtained in exchange for operating lease liability	$ 26,751

Nature of business

Nature of business	9 Months Ended
	Sep. 30, 2024
Nature of business
Nature of business	VOYAGER THERAPEUTICS INC. NOTE S TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS 1. Nature of business Voyager Therapeutics, Inc. (the “Company”) is a biotechnology company whose mission is to leverage the power of human genetics to modify the course of and ultimately cure neurological diseases. The Company’s pipeline includes programs for Alzheimer’s disease; amyotrophic lateral sclerosis; Parkinson’s disease, and multiple other diseases of the central nervous system. Many of the Company’s programs are derived from its TRACER™ adeno-associated virus (“AAV”) capsid discovery platform, which the Company has used to generate novel capsids (“TRACER Capsids”) and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of the Company’s programs are wholly-owned, and some are advancing with licensees and collaborators including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG, (“Novartis”); and Neurocrine Biosciences, Inc. (“Neurocrine”). The Company has a history of incurring annual net operating losses. As of September 30, 2024, the Company had an accumulated deficit of $291.7 million. The Company has not generated any product revenue and has financed its operations primarily through public offerings and private placements of its equity securities, funding from fees, option exercise payments, and milestone payments, and cost reimbursements associated with its prior and ongoing collaborations and license agreements. As of September 30, 2024, the Company had cash, cash equivalents, and marketable securities of $345.4 million. Based upon the Company’s current operating plans, the Company expects that its existing cash, cash equivalents, and marketable securities at September 30, 2024 to be sufficient to meet the Company’s planned operating expenses and capital expenditure requirements for at least twelve months from the issuance of these consolidated financial statements. There can be no assurance that the Company will be able to obtain additional debt or equity financing on terms acceptable to the Company or generate product revenue or revenue from collaboration partners, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition.

Summary of significant accounti

Summary of significant accounting policies and basis of presentation	9 Months Ended
	Sep. 30, 2024
Summary of significant accounting policies and basis of presentation
Summary of significant accounting policies and basis of presentation	2. Summary of significant accounting policies and basis of presentation Basis of Presentation The accompanying unaudited condensed consolidated financial statements of the Company have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. For further information, refer to the consolidated financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 as filed with the Securities and Exchange Commission (“SEC”) on February 28, 2024. These interim condensed consolidated financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the Company’s financial position and results of operations for the periods presented. Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification and Accounting Standards Updates of the Financial Accounting Standards Board. Principles of Consolidation The unaudited interim consolidated financial statements include the accounts of the Company and its wholly-owned subsidiary as disclosed in Note 2, under the heading “Summary of Significant Accounting Policies and Basis of Presentation,” within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023. Intercompany balances and transactions have been eliminated. Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, incremental borrowing rate for leases, accrued expenses, stock-based compensation expense, and income taxes. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Summary of Significant Accounting Policies There have been no changes in the Company's significant accounting policies as described in Note 2, “Summary of Significant Accounting Policies and Basis of Presentation,” within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the year ended December 31, 2023.

Fair value measurements

Fair value measurements	9 Months Ended
	Sep. 30, 2024
Fair value measurements
Fair value measurements	3. Fair value measurements Assets and liabilities measured at fair value on a recurring basis as of September 30, 2024 and December 31, 2023 are as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Quoted Prices ​ Significant ​ ​ ​ ​ ​ ​ ​ ​ in Active ​ Other ​ Significant ​ ​ ​ ​ ​ Markets for ​ Observable ​ Unobservable ​ ​ ​ ​ ​ Identical Assets ​ Inputs ​ Inputs ​ Assets Total (Level 1) (Level 2) (Level 3) September 30, 2024 ​ (in thousands) Money market funds included in cash and cash equivalents $ 106,492 $ 106,492 $ — $ — ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ 77,920 ​ 77,920 ​ — ​ — ​ U.S. Government agency securities ​ ​ 24,655 ​ ​ 24,655 ​ ​ — ​ ​ — ​ Certificates of deposit ​ ​ 5,461 ​ ​ — ​ ​ 5,461 ​ ​ — ​ Corporate bonds ​ ​ 102,083 ​ ​ — ​ ​ 102,083 ​ ​ — ​ Commercial paper ​ ​ 22,715 ​ ​ — ​ ​ 22,715 ​ ​ — ​ Total money market funds and marketable securities ​ $ 339,326 ​ $ 209,067 ​ $ 130,259 ​ $ — ​ December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds included in cash and cash equivalents $ 65,589 $ 65,589 $ — $ — ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ ​ 103,044 ​ ​ 103,044 ​ ​ — ​ ​ — ​ U.S. Government agency securities ​ ​ 31,075 ​ ​ 31,075 ​ ​ — ​ ​ — ​ Corporate bonds ​ ​ 23,970 ​ ​ — ​ ​ 23,970 ​ ​ — ​ Commercial paper ​ ​ 3,985 ​ — ​ ​ 3,985 ​ ​ — ​ Total money market funds and marketable securities ​ $ 227,663 ​ $ 199,708 ​ $ 27,955 ​ $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ The Company measures the fair value of money market funds, U.S. Treasury notes and U.S. Government agency securities based on quoted prices in active markets for identical securities. The Company measures the fair value of the Level 2 securities, certificates of deposit, corporate bonds and commercial paper based on recent trades of securities in active markets or based on quoted market prices of similar instruments and other significant inputs derived from or corroborated by observable market data.

Cash, cash equivalents, restric

Cash, cash equivalents, restricted cash, and available-for-sale marketable securities	9 Months Ended
	Sep. 30, 2024
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities	4. Cash, cash equivalents, restricted cash, and available-for-sale marketable securities Cash, cash equivalents, and marketable securities included the following at September 30, 2024 and December 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair ​ ​ Cost Gains Losses Value ​ ​ ​ (in thousands) ​ As of September 30, 2024 ​ ​ ​ ​ ​ Money market funds included in cash and cash equivalents ​ $ 106,492 ​ $ — ​ $ — ​ $ 106,492 ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ 77,846 ​ ​ 74 ​ ​ — ​ ​ 77,920 ​ U.S. Government agency securities ​ ​ 24,644 ​ ​ 13 ​ ​ (2) ​ ​ 24,655 ​ Certificates of deposit ​ ​ 5,449 ​ ​ 12 ​ ​ — ​ ​ 5,461 ​ Corporate bonds ​ ​ 102,077 ​ ​ 55 ​ ​ (49) ​ ​ 102,083 ​ Commercial paper ​ ​ 22,724 ​ ​ 4 ​ ​ (13) ​ ​ 22,715 ​ Total money market funds and marketable securities ​ $ 339,232 ​ $ 158 ​ $ (64) ​ $ 339,326 ​ As of December 31, 2023 ​ ​ ​ ​ ​ Money market funds included in cash and cash equivalents ​ $ 65,589 ​ $ — ​ $ — ​ $ 65,589 ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ 102,966 ​ ​ 81 ​ ​ (3) ​ 103,044 ​ U.S. Government agency securities ​ ​ 31,068 ​ ​ 10 ​ ​ (3) ​ ​ 31,075 ​ Corporate bonds ​ ​ 23,975 ​ ​ 2 ​ ​ (7) ​ ​ 23,970 ​ Commercial paper ​ ​ 3,985 ​ ​ — ​ ​ — ​ ​ 3,985 ​ Total money market funds and marketable securities ​ $ 227,583 ​ $ 93 ​ $ (13) ​ $ 227,663 ​ ​ All of the Company’s marketable securities as of September 30, 2024 have a contractual maturity of one year or less. The Company reviews investments whenever the fair value of an investment is less than the amortized cost and evidence indicates that an investment’s carrying amount is not recoverable within a reasonable period of time. In connection with these investments, the Company evaluates whether the decline in fair value has resulted from credit losses or other factors, considering the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security, among other factors. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security is compared to the amortized cost basis of the security. If the present value of cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss on the condensed consolidated balance sheet, limited by the amount that the fair value is less than the amortized cost basis. Any impairment that is not related to credit is recognized in other comprehensive (loss) income. Changes in the allowance for credit losses are recorded as a provision for (or reversal of) credit loss expense in general and administrative expenses within the condensed consolidated statement of operations. Losses are charged against the allowance when the Company believes the uncollectability of an available-for-sale security is confirmed or when either of the criteria regarding intent or requirement to sell is met. The Company held $96.0 million and $44.2 million in marketable securities that were in an unrealized loss position as of September 30, 2024 and December 31, 2023, respectively. The unrealized losses at September 30, 2024 and December 31, 2023 were attributable to changes in interest rates and do not represent credit losses. The Company does not intend to sell these securities and it is not more likely than not that it will be required to sell them before recovery of their amortized cost basis. The following table provides a reconciliation of cash, cash equivalents, and restricted cash within the condensed consolidated balance sheets that sum to the total of the same such amounts shown in the condensed consolidated statements of cash flows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of September 30, ​ As of December 31, ​ ​ ​ 2024 2023 ​ ​ (in thousands) ​ Cash and cash equivalents ​ $ 112,525 ​ $ 68,802 ​ Restricted cash included in deposits and other non-current assets ​ ​ 2,874 ​ ​ 1,593 ​ Total cash, cash equivalents, and restricted cash ​ $ 115,399 ​ $ 70,395 ​ ​

Accrued expenses

Accrued expenses	9 Months Ended
	Sep. 30, 2024
Accrued expenses
Accrued expenses	5. Accrued expenses Accrued expenses as of September 30, 2024 and December 31, 2023 consist of the following: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of September 30, ​ As of December 31, ​ ​ 2024 2023 ​ ​ (in thousands) ​ Employee compensation costs ​ $ 5,927 ​ $ 6,614 ​ Research and development costs ​ ​ 3,972 ​ ​ 5,225 ​ Accrued goods and services ​ ​ 530 ​ ​ 4,229 ​ Professional services ​ 1,013 ​ 755 ​ Total ​ $ 11,442 ​ $ 16,823 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​

Lease obligation

Lease obligation	9 Months Ended
	Sep. 30, 2024
Lease obligation
Lease obligation	6. Lease obligation Operating Leases As of September 30, 2024, the Company has a lease for laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts through January 31, 2031 and a lease for additional office and laboratory space at 64 Sidney Street in Cambridge, Massachusetts (the “Cambridge Facility”) through November 30, 2026. On August 11, 2023, the Company entered into a first amendment (the “First Amendment”) to its existing lease for laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts, pursuant to which the Company agreed to lease approximately 61,307 square feet of additional office and laboratory space through January 31, 2031. The Company received $1.8 million of leasehold improvement incentives associated with the First Amendment. The Company gained control of the space on February 1, 2024 and recorded a $26.7 million right-of-use asset and a $26.7 million operating lease liability, accordingly, which reflect the leasehold improvement incentive. The Company’s lease agreements require the Company to maintain a cash deposit or irrevocable letter of credit in the aggregate amount of $2.9 million payable to its landlords as security for the performance of its obligations under the leases. These amounts are recorded as restricted cash and are included in deposits and other non-current assets in the accompanying condensed consolidated balance sheets. In June 2024, the Company vacated its leased office and laboratory space in Cambridge, Massachusetts. The Company recorded an impairment charge of $2.8 million to operating expenses during the second quarter of 2024 as a result of the carrying value of the leased office and laboratory space asset group exceeding the undiscounted cash flows projected from a planned sublease of the facility. The impairment charge reduced the carrying value of the leased office and laboratory space asset group by $2.8 million. On August 2, 2024, the Company executed the sublease of the Cambridge Facility. Payments received under the sublease are included in other income in the condensed consolidated statements of operations and comprehensive (loss) income. During the three and nine months ended September 30, 2024, the Company recognized $0.2 million of other income from its sublease of the Cambridge Facility. During the three and nine months ended September 30, 2024, the Company incurred lease expenses of $1.9 million and $8.0 million, respectively. During the three and nine months ended September 30, 2023, the Company incurred lease expenses of $0.9 million and $2.7 million, respectively, for operating leases. As of September 30, 2024, the weighted average remaining lease term was 4.9 years and the weighted average incremental borrowing rate used to determine the operating lease liability was 6.8%. ​

Commitments, contingencies and

Commitments, contingencies and other liabilities	9 Months Ended
	Sep. 30, 2024
Commitments, contingencies and other liabilities
Commitments, contingencies and other liabilities	7. Commitments, contingencies and other liabilities As of September 30, 2024 and December 31, 2023, other current and non-current liabilities consisted of the following: ​ ​ ​ ​ ​ ​ ​ ​ ​ As of September 30, ​ As of December 31, ​ ​ 2024 2023 ​ ​ (in thousands) ​ Other current liabilities ​ ​ ​ ​ ​ ​ Lease liability ​ 6,979 ​ ​ 3,200 ​ Total other current liabilities $ 6,979 ​ $ 3,200 ​ ​ ​ ​ ​ ​ ​ ​ Other non-current liabilities ​ ​ ​ ​ ​ ​ Lease liability $ 38,368 ​ $ 17,093 ​ Other ​ 1,000 ​ ​ 1,001 ​ Total other non-current liabilities $ 39,368 ​ $ 18,094 ​ ​ Other Agreements ​ ​ Litigation ​

Significant agreements

Significant agreements	9 Months Ended
	Sep. 30, 2024
Significant agreements
Significant agreements	8. Significant agreements The Company’s significant agreements are described in Note 9 of the December 31, 2023 consolidated financial statements included in its Annual Report on Form 10-K for the year ended December 31, 2023. During the three and nine months ended September 30, 2024, there were no material changes to the terms of the Company’s collaboration agreements with Neurocrine and the Company did not enter into any new collaboration or license agreements. On September 3, 2024, the Company entered into an amendment to its existing option and license agreement with Novartis dated March 4, 2022 (the “2022 Novartis Option and License Agreement”). The Company recorded collaboration revenue of $24.6 million and $4.6 million during the three months ended September 30, 2024 and 2023, respectively. The Company recorded collaboration revenue of $73.7 million and $159.9 million during the nine months ended September 30, 2024 and 2023, respectively. 2022 Novartis Option and License Agreement On September 3, 2024 (the “Amendment Effective Date”), the Company and Novartis entered into an amendment (the “Novartis Amendment”) to the 2022 Novartis Option and License Agreement. Pursuant to the Novartis Amendment, the Company and Novartis agreed to amend the 2022 Novartis Option and License Agreement to incorporate the grant from the Company to Novartis of an additional license (the “Novartis Direct License”) to a capsid discovered through the Company’s TRACER discovery platform (the “Novartis Direct Licensed Capsid”) for exclusive use with a certain gene (the “Novartis Direct License Target”) to develop and commercialize the Novartis Direct Licensed Capsid as incorporated into products containing a payload directed to the Novartis Direct License Target (“Novartis Direct Licensed Products”). As a result of the Novartis Amendment, the Novartis Direct License Target is now deemed a Licensed Target under the 2022 Novartis Option and License Agreement, as such term is defined therein, and the Novartis Direct License is subject to all other terms and conditions applicable to licenses granted to Novartis under the 2022 Novartis Option and License Agreement. Novartis agreed to pay to the Company a one-time fee of $15.0 million in consideration for the rights granted under the Novartis Amendment, which was received in October 2024. The Company evaluated the Novartis Amendment under ASC 606 and recorded $15.0 million as collaboration revenue in the three months ended September 30, 2024 since the consideration reflected the standalone selling price of the license that was delivered on the Novartis Amendment date, and there were no remaining undelivered performance obligations under the original 2022 Novartis Option and License Agreement. The Company is eligible to receive specified development, regulatory, and commercialization milestone payments of up to an aggregate of $130.0 million for the first Novartis Direct Licensed Product to achieve the corresponding milestone. On a Novartis Direct Licensed Product-by-Novartis Direct Licensed Product basis, the Company is also eligible to receive (a) specified sales milestone payments of up to an aggregate of $175.0 million per Novartis Direct Licensed Product and (b) tiered, escalating royalties in the mid- to high-single-digit percentages of annual net sales of each Novartis Direct Licensed Product. ​ 2023 Neurocrine Collaboration Agreement In April 2024, the Company announced that the joint steering committee with Neurocrine selected a development candidate for the glucocerebrosidase 1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases (the “GBA1 program”) under the collaboration and license agreement with Neurocrine entered into in January 2023 (the “2023 Neurocrine Collaboration Agreement”). The joint steering committee selection of a development candidate for the GBA1 Program triggered a $3.0 million milestone payment to the Company. The Company recorded the $3.0 million as collaboration revenue during the second quarter of 2024. In September 2024, the Company announced that the joint steering committee with Neurocrine selected a development candidate in a gene therapy program for the potential treatment of an undisclosed neurological disease under the 2023 Neurocrine Collaboration Agreement, which triggered a $3.0 million milestone payment to the Company which was received in October 2024. The Company included the $3.0 million that had previously been constrained in the transaction price allocated to the program’s performance obligation in the three months ended September 30, 2024, accordingly, which resulted in a cumulative catch-up adjustment to collaboration revenue of approximately $2.0 million. ​ 2023 Novartis Stock Purchase Agreement ​ Under the stock purchase agreement entered into in December 2023 (the “2023 Novartis Stock Purchase Agreement”), Novartis purchased 2,145,002 shares of common stock of the Company (the “Novartis Shares”) for an aggregate purchase price of approximately $20.0 million. The issuance of the Novartis Shares to Novartis pursuant to the 2023 Novartis Stock Purchase Agreement in January 2024 resulted in a premium of $0.7 million. The premium was allocated to the development and commercialization licenses granted to Novartis for two programs pursuant to the license and collaboration agreement with Novartis entered into in December 2023 and was recognized as collaboration revenue during the first quarter of 2024, upon the issuance of the Novartis Shares under the 2023 Novartis Stock Purchase Agreement. ​ 2019 Neurocrine Collaboration Agreement ​ In February 2024, the Company announced that the joint steering committee with Neurocrine selected a lead development candidate for the gene therapy program for Friedreich’s ataxia (the “FA Program”) under the collaboration and license agreement with Neurocrine entered into in January 2019 (the “2019 Neurocrine Collaboration Agreement”), which triggered a $5.0 million milestone payment to the Company that was received in the first quarter of 2024. The Company included the $5.0 million that had previously been constrained in the transaction price allocated to the FA Program performance obligation in the three months ended March 31, 2024, accordingly, which resulted in a cumulative catch-up adjustment to collaboration revenue of $4.4 million. ​ Related Party Collaboration Receivable ​ The following table presents changes in the balances of the Company’s related party collaboration receivable and contract liabilities for the 2023 Neurocrine Collaboration Agreement and the 2019 Neurocrine Collaboration Agreement during the nine months ended September 30, 2024: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at ​ ​ ​ ​ Balance at ​ ​ December 31, 2023 ​ Additions ​ Deductions ​ September 30, 2024 ​ ​ ​ ( in thousands) Related party collaboration receivables ​ $ 3,341 ​ $ 11,895 ​ $ (11,522) ​ $ 3,714 Contract liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Deferred revenue ​ $ 75,240 ​ $ 1,592 ​ $ (42,050) ​ $ 34,782 ​ The change in the related party collaboration receivable balance for the nine months ended September 30, 2024 is primarily driven by amounts owed to the Company for research and development services provided, offset by amounts collected during the period, for the 2023 and 2019 Neurocrine Collaboration Agreements. Deferred revenue activity for the period includes the recording of $1.6 million of deferred revenue during the nine months ended September 30, 2024 related to fixed transaction price allocation increases, offset by $42.1 million of collaboration revenue recognized on the proportional performance model during the period for the 2023 and 2019 Neurocrine Collaboration Agreements, which is classified as either current or non-current in the accompanying consolidated balance sheet based on the period the services are expected to be delivered.

Stock-based compensation

Stock-based compensation	9 Months Ended
	Sep. 30, 2024
Stock-based compensation
Stock-based compensation	9. Stock-based compensation Stock-Based Compensation Expense Total compensation cost recognized for all stock-based compensation awards in the condensed consolidated statements of operations and comprehensive (loss) income was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Nine Months Ended ​ ​ ​ September 30, September 30, ​ 2024 2023 2024 2023 ​ ​ (in thousands) Research and development ​ $ 1,590 ​ $ 919 ​ $ 4,495 ​ $ 2,472 ​ General and administrative ​ 1,844 ​ 1,959 ​ 6,553 ​ 5,636 ​ Total stock-based compensation expense ​ $ 3,434 ​ $ 2,878 ​ $ 11,048 ​ $ 8,108 ​ ​ Stock-based compensation expense by type of award included within the condensed consolidated statements of operations and comprehensive (loss) income was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Nine Months Ended ​ ​ ​ September 30, ​ September 30, ​ ​ 2024 2023 ​ 2024 2023 ​ ​ ​ (in thousands) ​ Stock options ​ $ 2,373 ​ $ 2,021 ​ $ 7,357 ​ $ 5,587 ​ Restricted stock awards and units ​ ​ 983 ​ ​ 774 ​ ​ 3,454 ​ ​ 2,339 ​ Employee stock purchase plan awards ​ 78 ​ 83 ​ ​ 237 ​ ​ 182 ​ Total stock-based compensation expense ​ $ 3,434 ​ $ 2,878 ​ $ 11,048 ​ $ 8,108 ​ ​ Restricted Stock Units A summary of the status of and changes in unvested restricted stock unit activity under the Company’s equity award plans for the nine months ended September 30, 2024 was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted ​ ​ ​ ​ ​ Average ​ ​ ​ ​ ​ Grant Date ​ ​ ​ ​ ​ Fair Value ​ ​ Units Per Unit ​ Unvested restricted stock units as of December 31, 2023 1,370,897 ​ $ 6.65 ​ Granted 867,558 ​ $ 8.15 ​ Vested (423,362) ​ $ 6.23 ​ Forfeited (198,076) ​ $ 6.68 ​ Unvested restricted stock units as of September 30, 2024 1,617,017 ​ $ 7.56 ​ ​ Stock-based compensation of restricted stock units is based on the fair value of the Company’s common stock on the date of grant and is recognized over the vesting period. All of the restricted stock units granted in the nine months ended September 30, 2024 vest in equal amounts, annually over three years. The stock-based compensation expense related to restricted stock units was $1.0 million and $3.5 million for the three and nine months ended September 30, 2024, respectively. The stock-based compensation expense related to restricted stock units was $0.8 million and $2.3 million for the three and nine months ended September 30, 2023, respectively. As of September 30, 2024, the Company had unrecognized stock-based compensation expense related to its unvested restricted stock units of $9.1 million, which is expected to be recognized over the remaining average vesting period of 2.1 years. Stock Options The following is a summary of stock option activity for the nine months ended September 30, 2024: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted Remaining Aggregate ​ ​ ​ ​ ​ Average ​ Contractual ​ Intrinsic ​ ​ ​ ​ ​ Exercise ​ Life ​ Value ​ ​ Shares Price (in years) (in thousands) ​ Outstanding at December 31, 2023 7,425,444 ​ $ 8.52 ​ ​ ​ ​ ​ ​ Granted 2,325,226 ​ $ 7.79 ​ ​ ​ ​ ​ ​ Exercised (86,100) ​ $ 4.11 ​ ​ ​ ​ ​ ​ Cancelled or forfeited (793,242) ​ $ 11.69 ​ ​ ​ ​ ​ ​ Outstanding at September 30, 2024 8,871,328 ​ $ 8.08 7.5 ​ ​ 3,756 ​ Exercisable at September 30, 2024 4,604,321 ​ $ 8.51 6.4 ​ ​ 2,777 ​ ​ As of September 30, 2024, the Company had unrecognized stock-based compensation expense related to its unvested stock options of $20.1 million which is expected to be recognized over the remaining weighted-average vesting period of 2.7 years.

Net (loss) income per share

Net (loss) income per share	9 Months Ended
	Sep. 30, 2024
Net (loss) income per share
Net (loss) income per share	10. Net (loss) income per share The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net (loss) income per share because to include them would be anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended September 30, ​ ​ Nine Months Ended September 30, ​ ​ ​ 2024 ​ 2023 ​ 2024 ​ 2023 ​ Unvested restricted common stock awards ​ — ​ ​ 22,500 ​ — ​ ​ 22,500 ​ Unvested restricted common stock units ​ ​ 1,617,017 ​ ​ 1,312,727 ​ ​ 1,617,017 ​ ​ 714,164 ​ Outstanding stock options ​ 8,871,328 ​ ​ 7,745,593 ​ 8,871,328 ​ ​ 6,695,548 ​ Total ​ 10,488,345 ​ ​ 9,080,820 ​ 10,488,345 ​ ​ 7,432,212 ​ ​ Basic and diluted and diluted weighted-average shares outstanding are as follows for the three and nine months ended September 30, 2024 and 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended September 30, ​ ​ Nine Months Ended September 30, ​ ​ ​ 2024 ​ ​ 2023 ​ ​ 2024 ​ ​ 2023 ​ ​ ​ ​ ​ ​ ​ Numerator: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net (loss) income (in thousands) ​ $ (9,044) ​ $ (25,901) ​ $ (30,515) ​ $ 75,935 Denominator for basic net (loss) income per share: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted average shares outstanding-basic ​ ​ 57,851,110 ​ ​ 43,864,838 ​ ​ 57,564,413 ​ ​ 40,962,116 Denominator for diluted net (loss) income per share: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted average shares outstanding-basic ​ ​ 57,851,110 ​ ​ 43,864,838 ​ ​ 57,564,413 ​ ​ 40,962,116 Common stock options and restricted stock units ​ ​ — ​ ​ — ​ ​ — ​ ​ 1,648,608 Weighted average shares outstanding-diluted ​ ​ 57,851,110 ​ ​ 43,864,838 ​ ​ 57,564,413 ​ ​ 42,610,724 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net (loss) income per share, basic: ​ $ (0.16) ​ $ (0.59) ​ $ (0.53) ​ $ 1.85 Net (loss) income per share, diluted: ​ $ (0.16) ​ $ (0.59) ​ $ (0.53) ​ $ 1.78 ​ ​ ​ The pre ghted average shares outstanding for the three and nine months ended September 30, 2024.

Underwritten public offering

Underwritten public offering	9 Months Ended
	Sep. 30, 2024
Underwritten public offering
Underwritten public offering	11. Underwritten public offering ​ On January 4, 2024, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with Citigroup Global Markets Inc. and Guggenheim Securities, LLC, as representatives of the several underwriters named therein (the “Underwriters”), relating to an underwritten public offering of 7,777,778 shares of the Company’s common stock, par value $0.001 per share, and, in lieu of common stock to certain investors, pre-funded warrants (the “Pre-Funded Warrants”) to purchase up to 3,333,333 shares of common stock. The Underwriters agreed to purchase the Company’s stock from the Company pursuant to the Underwriting Agreement at a price of $8.46 and the Pre-Funded Warrants from the Company pursuant to the Underwriting Agreement at a price of $8.459 per share underlying each Pre-Funded Warrant. ​ ​

Related party transactions

Related party transactions	9 Months Ended
	Sep. 30, 2024
Related party transactions
Related party transactions	12. Related-party transactions During the three and nine months ended September 30, 2024, the Company received scientific advisory board and other scientific advisory services from Dinah Sah, Ph.D., the Company’s former Chief Scientific Officer. The total amount of fees paid to Dr. Sah for services provided during the three and nine months ended September 30, 2024, was $150,000 and $450,000, respectively. The total amount of fees paid to Dr. Sah for services provided during the three and nine months ended September 30, 2023 was $157,500 and $541,300, respectively. During the second quarter of 2023, the Company and Dr. Sah agreed to a fee of $50,000 per month for advisory services from Dr. Sah pursuant to an amendment to the Company’s consulting agreement with Dr. Sah that became effective in June 2023. Under each of the Company’s collaboration agreements with Neurocrine, the Company and Neurocrine have agreed to conduct research, development, and commercialization activities for certain of the Company’s AAV gene therapy product candidates. Amounts due from Neurocrine are reflected as related party collaboration receivables. As of September 30, 2024, the Company had approximately $3.3 million in related party collaboration receivables associated with the 2023 Neurocrine Collaboration Agreement and approximately $0.4 million in related party collaboration receivables associated with the 2019 Neurocrine Collaboration Agreement.

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (9,044)	$ (10,141)	$ (11,330)	$ (25,901)	$ (22,208)	$ 124,044	$ (30,515)	$ 75,935

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
	Sep. 30, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	(c) Director and Officer Trading Arrangements ​ A portion of the compensation of our directors and officers (as defined in Rule 16a-1(f) under the Securities Exchange Act of 1934, as amended, or the Exchange Act,” is in the form of equity awards and, from time to time, directors and officers engage in open-market transactions with respect to the securities acquired pursuant to such equity awards or our other securities, including to satisfy tax withholding obligations when equity awards vest or are exercised, for diversification or other personal reasons. ​ Transactions in our securities by directors and officers are required to be made in accordance with our insider trading policy, which requires that the transactions be in accordance with applicable U.S. federal securities laws that prohibit trading while in possession of material nonpublic information. Rule 10b5-1 under the Exchange Act provides an affirmative defense that enables directors and officers to prearrange transactions in our securities in a manner that avoids concerns about initiating transactions while in possession of material nonpublic information. ​ The following table describes, for the quarterly period covered by this report, each trading arrangement for the sale or purchase of our securities adopted or terminated by our directors and officers that is either (1) a contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c), or a Rule 10b5-1 trading arrangement, or (2) a non-Rule 10b5-1 trading arrangement, as defined in Item 408(c) of Regulation S-K. ​ ​ ​ ​ ​ ​ ​ ​ ​ Type of Nature of Duration of Trading Aggregate ​ Action Taken Trading Trading Trading Number of Name (Title) (Date of Action) Arrangement Arrangement Arrangement Securities Todd Carter, Ph.D. Adoption Rule 10b5-1 trading arrangement for exercises of options and sales of shares Sale Until August 15, 2025, or such earlier date upon which all transactions are completed or expire without execution Up to 79,000 shares ​
Todd Carter, Ph.D.
Trading Arrangements, by Individual
Name	Todd Carter, Ph.D.
Title	Chief Scientific Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	September 9, 2024
Aggregate Available	79,000

Summary of significant accoun_2

Summary of significant accounting policies and basis of presentation (Policies)	9 Months Ended
	Sep. 30, 2024
Summary of significant accounting policies and basis of presentation
Basis of Presentation	Basis of Presentation The accompanying unaudited condensed consolidated financial statements of the Company have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. For further information, refer to the consolidated financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 as filed with the Securities and Exchange Commission (“SEC”) on February 28, 2024. These interim condensed consolidated financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the Company’s financial position and results of operations for the periods presented. Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification and Accounting Standards Updates of the Financial Accounting Standards Board.
Principles of Consolidation	Principles of Consolidation The unaudited interim consolidated financial statements include the accounts of the Company and its wholly-owned subsidiary as disclosed in Note 2, under the heading “Summary of Significant Accounting Policies and Basis of Presentation,” within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023. Intercompany balances and transactions have been eliminated.
Use of Estimates	Use of Estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, incremental borrowing rate for leases, accrued expenses, stock-based compensation expense, and income taxes. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions.
Summary of Significant Accounting Policies	Summary of Significant Accounting Policies There have been no changes in the Company's significant accounting policies as described in Note 2, “Summary of Significant Accounting Policies and Basis of Presentation,” within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the year ended December 31, 2023.

Fair value measurements (Tables

Fair value measurements (Tables)	9 Months Ended
	Sep. 30, 2024
Fair value measurements
Schedule of assets and liabilities measured at fair value on a recurring basis	Assets and liabilities measured at fair value on a recurring basis as of September 30, 2024 and December 31, 2023 are as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Quoted Prices ​ Significant ​ ​ ​ ​ ​ ​ ​ ​ in Active ​ Other ​ Significant ​ ​ ​ ​ ​ Markets for ​ Observable ​ Unobservable ​ ​ ​ ​ ​ Identical Assets ​ Inputs ​ Inputs ​ Assets Total (Level 1) (Level 2) (Level 3) September 30, 2024 ​ (in thousands) Money market funds included in cash and cash equivalents $ 106,492 $ 106,492 $ — $ — ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ 77,920 ​ 77,920 ​ — ​ — ​ U.S. Government agency securities ​ ​ 24,655 ​ ​ 24,655 ​ ​ — ​ ​ — ​ Certificates of deposit ​ ​ 5,461 ​ ​ — ​ ​ 5,461 ​ ​ — ​ Corporate bonds ​ ​ 102,083 ​ ​ — ​ ​ 102,083 ​ ​ — ​ Commercial paper ​ ​ 22,715 ​ ​ — ​ ​ 22,715 ​ ​ — ​ Total money market funds and marketable securities ​ $ 339,326 ​ $ 209,067 ​ $ 130,259 ​ $ — ​ December 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds included in cash and cash equivalents $ 65,589 $ 65,589 $ — $ — ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ ​ 103,044 ​ ​ 103,044 ​ ​ — ​ ​ — ​ U.S. Government agency securities ​ ​ 31,075 ​ ​ 31,075 ​ ​ — ​ ​ — ​ Corporate bonds ​ ​ 23,970 ​ ​ — ​ ​ 23,970 ​ ​ — ​ Commercial paper ​ ​ 3,985 ​ — ​ ​ 3,985 ​ ​ — ​ Total money market funds and marketable securities ​ $ 227,663 ​ $ 199,708 ​ $ 27,955 ​ $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​

Cash, cash equivalents, restr_2

Cash, cash equivalents, restricted cash, and available-for-sale marketable securities (Tables)	9 Months Ended
	Sep. 30, 2024
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities
Schedule of money market funds and marketable securities	Cash, cash equivalents, and marketable securities included the following at September 30, 2024 and December 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair ​ ​ Cost Gains Losses Value ​ ​ ​ (in thousands) ​ As of September 30, 2024 ​ ​ ​ ​ ​ Money market funds included in cash and cash equivalents ​ $ 106,492 ​ $ — ​ $ — ​ $ 106,492 ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ 77,846 ​ ​ 74 ​ ​ — ​ ​ 77,920 ​ U.S. Government agency securities ​ ​ 24,644 ​ ​ 13 ​ ​ (2) ​ ​ 24,655 ​ Certificates of deposit ​ ​ 5,449 ​ ​ 12 ​ ​ — ​ ​ 5,461 ​ Corporate bonds ​ ​ 102,077 ​ ​ 55 ​ ​ (49) ​ ​ 102,083 ​ Commercial paper ​ ​ 22,724 ​ ​ 4 ​ ​ (13) ​ ​ 22,715 ​ Total money market funds and marketable securities ​ $ 339,232 ​ $ 158 ​ $ (64) ​ $ 339,326 ​ As of December 31, 2023 ​ ​ ​ ​ ​ Money market funds included in cash and cash equivalents ​ $ 65,589 ​ $ — ​ $ — ​ $ 65,589 ​ Marketable securities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. Treasury notes ​ 102,966 ​ ​ 81 ​ ​ (3) ​ 103,044 ​ U.S. Government agency securities ​ ​ 31,068 ​ ​ 10 ​ ​ (3) ​ ​ 31,075 ​ Corporate bonds ​ ​ 23,975 ​ ​ 2 ​ ​ (7) ​ ​ 23,970 ​ Commercial paper ​ ​ 3,985 ​ ​ — ​ ​ — ​ ​ 3,985 ​ Total money market funds and marketable securities ​ $ 227,583 ​ $ 93 ​ $ (13) ​ $ 227,663 ​
Reconciliation of cash, cash equivalents, and restricted cash	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of September 30, ​ As of December 31, ​ ​ ​ 2024 2023 ​ ​ (in thousands) ​ Cash and cash equivalents ​ $ 112,525 ​ $ 68,802 ​ Restricted cash included in deposits and other non-current assets ​ ​ 2,874 ​ ​ 1,593 ​ Total cash, cash equivalents, and restricted cash ​ $ 115,399 ​ $ 70,395 ​ ​

Accrued expenses (Tables)

Accrued expenses (Tables)	9 Months Ended
	Sep. 30, 2024
Accrued expenses
Schedule of accrued expenses	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of September 30, ​ As of December 31, ​ ​ 2024 2023 ​ ​ (in thousands) ​ Employee compensation costs ​ $ 5,927 ​ $ 6,614 ​ Research and development costs ​ ​ 3,972 ​ ​ 5,225 ​ Accrued goods and services ​ ​ 530 ​ ​ 4,229 ​ Professional services ​ 1,013 ​ 755 ​ Total ​ $ 11,442 ​ $ 16,823 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​

Commitments, contingencies an_2

Commitments, contingencies and other liabilities (Tables)	9 Months Ended
	Sep. 30, 2024
Commitments, contingencies and other liabilities
Summary of other current and non-current liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ As of September 30, ​ As of December 31, ​ ​ 2024 2023 ​ ​ (in thousands) ​ Other current liabilities ​ ​ ​ ​ ​ ​ Lease liability ​ 6,979 ​ ​ 3,200 ​ Total other current liabilities $ 6,979 ​ $ 3,200 ​ ​ ​ ​ ​ ​ ​ ​ Other non-current liabilities ​ ​ ​ ​ ​ ​ Lease liability $ 38,368 ​ $ 17,093 ​ Other ​ 1,000 ​ ​ 1,001 ​ Total other non-current liabilities $ 39,368 ​ $ 18,094 ​

Significant agreements (Tables)

Significant agreements (Tables)	9 Months Ended
	Sep. 30, 2024
2023 Neurocrine Collaborative Agreement and 2019 Neurocrine Collaborative Agreement
Schedule of collaboration receivables and contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at ​ ​ ​ ​ Balance at ​ ​ December 31, 2023 ​ Additions ​ Deductions ​ September 30, 2024 ​ ​ ​ ( in thousands) Related party collaboration receivables ​ $ 3,341 ​ $ 11,895 ​ $ (11,522) ​ $ 3,714 Contract liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Deferred revenue ​ $ 75,240 ​ $ 1,592 ​ $ (42,050) ​ $ 34,782

Stock-based compensation (Table

Stock-based compensation (Tables)	9 Months Ended
	Sep. 30, 2024
Stock-based compensation
Compensation cost recognized for all stock-based compensation awards	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Nine Months Ended ​ ​ ​ September 30, September 30, ​ 2024 2023 2024 2023 ​ ​ (in thousands) Research and development ​ $ 1,590 ​ $ 919 ​ $ 4,495 ​ $ 2,472 ​ General and administrative ​ 1,844 ​ 1,959 ​ 6,553 ​ 5,636 ​ Total stock-based compensation expense ​ $ 3,434 ​ $ 2,878 ​ $ 11,048 ​ $ 8,108 ​
Summary of stock-based compensation expense by type of award	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Nine Months Ended ​ ​ ​ September 30, ​ September 30, ​ ​ 2024 2023 ​ 2024 2023 ​ ​ ​ (in thousands) ​ Stock options ​ $ 2,373 ​ $ 2,021 ​ $ 7,357 ​ $ 5,587 ​ Restricted stock awards and units ​ ​ 983 ​ ​ 774 ​ ​ 3,454 ​ ​ 2,339 ​ Employee stock purchase plan awards ​ 78 ​ 83 ​ ​ 237 ​ ​ 182 ​ Total stock-based compensation expense ​ $ 3,434 ​ $ 2,878 ​ $ 11,048 ​ $ 8,108 ​
Summary of status and changes in unvested restricted stock	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted ​ ​ ​ ​ ​ Average ​ ​ ​ ​ ​ Grant Date ​ ​ ​ ​ ​ Fair Value ​ ​ Units Per Unit ​ Unvested restricted stock units as of December 31, 2023 1,370,897 ​ $ 6.65 ​ Granted 867,558 ​ $ 8.15 ​ Vested (423,362) ​ $ 6.23 ​ Forfeited (198,076) ​ $ 6.68 ​ Unvested restricted stock units as of September 30, 2024 1,617,017 ​ $ 7.56 ​
Summary of stock option activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted Remaining Aggregate ​ ​ ​ ​ ​ Average ​ Contractual ​ Intrinsic ​ ​ ​ ​ ​ Exercise ​ Life ​ Value ​ ​ Shares Price (in years) (in thousands) ​ Outstanding at December 31, 2023 7,425,444 ​ $ 8.52 ​ ​ ​ ​ ​ ​ Granted 2,325,226 ​ $ 7.79 ​ ​ ​ ​ ​ ​ Exercised (86,100) ​ $ 4.11 ​ ​ ​ ​ ​ ​ Cancelled or forfeited (793,242) ​ $ 11.69 ​ ​ ​ ​ ​ ​ Outstanding at September 30, 2024 8,871,328 ​ $ 8.08 7.5 ​ ​ 3,756 ​ Exercisable at September 30, 2024 4,604,321 ​ $ 8.51 6.4 ​ ​ 2,777 ​

Net (loss) income per share (Ta

Net (loss) income per share (Tables)	9 Months Ended
	Sep. 30, 2024
Net (loss) income per share
Outstanding potentially dilutive securities excluded in the calculation of diluted net (loss) income per share	The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net (loss) income per share because to include them would be anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended September 30, ​ ​ Nine Months Ended September 30, ​ ​ ​ 2024 ​ 2023 ​ 2024 ​ 2023 ​ Unvested restricted common stock awards ​ — ​ ​ 22,500 ​ — ​ ​ 22,500 ​ Unvested restricted common stock units ​ ​ 1,617,017 ​ ​ 1,312,727 ​ ​ 1,617,017 ​ ​ 714,164 ​ Outstanding stock options ​ 8,871,328 ​ ​ 7,745,593 ​ 8,871,328 ​ ​ 6,695,548 ​ Total ​ 10,488,345 ​ ​ 9,080,820 ​ 10,488,345 ​ ​ 7,432,212 ​
Schedule of weighted average number of shares, basic and diluted	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended September 30, ​ ​ Nine Months Ended September 30, ​ ​ ​ 2024 ​ ​ 2023 ​ ​ 2024 ​ ​ 2023 ​ ​ ​ ​ ​ ​ ​ Numerator: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net (loss) income (in thousands) ​ $ (9,044) ​ $ (25,901) ​ $ (30,515) ​ $ 75,935 Denominator for basic net (loss) income per share: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted average shares outstanding-basic ​ ​ 57,851,110 ​ ​ 43,864,838 ​ ​ 57,564,413 ​ ​ 40,962,116 Denominator for diluted net (loss) income per share: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted average shares outstanding-basic ​ ​ 57,851,110 ​ ​ 43,864,838 ​ ​ 57,564,413 ​ ​ 40,962,116 Common stock options and restricted stock units ​ ​ — ​ ​ — ​ ​ — ​ ​ 1,648,608 Weighted average shares outstanding-diluted ​ ​ 57,851,110 ​ ​ 43,864,838 ​ ​ 57,564,413 ​ ​ 42,610,724 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net (loss) income per share, basic: ​ $ (0.16) ​ $ (0.59) ​ $ (0.53) ​ $ 1.85 Net (loss) income per share, diluted: ​ $ (0.16) ​ $ (0.59) ​ $ (0.53) ​ $ 1.78

Nature of business (Details)

Nature of business (Details) - USD ($) $ in Thousands	Sep. 03, 2024	Sep. 30, 2024	Dec. 31, 2023
Accumulated deficit		$ (291,697)	$ (261,182)
Cash, cash equivalents, and marketable securities		$ 345,400
Novartis Pharma, AG | Option and License Agreement
Upfront payment	$ 15,000

Fair value measurements - Asset

Fair value measurements - Assets and liabilities measured on a recurring basis - (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Assets, Fair Value Disclosure [Abstract]
Cash and cash equivalents	$ 112,525	$ 68,802
Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Total money market funds and marketable securities	339,326	227,663
Level 1 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Total money market funds and marketable securities	209,067	199,708
Level 2 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Total money market funds and marketable securities	130,259	27,955
U.S. Treasury notes | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	77,920	103,044
U.S. Treasury notes | Level 1 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	77,920	103,044
U. S. Government agency securities | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	24,655	31,075
U. S. Government agency securities | Level 1 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	24,655	31,075
Corporate bonds | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	102,083	23,970
Corporate bonds | Level 2 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	102,083	23,970
Commercial paper | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	22,715	3,985
Commercial paper | Level 2 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	22,715	3,985
Money market funds
Assets, Fair Value Disclosure [Abstract]
Cash and cash equivalents	106,492	65,589
Money market funds | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Cash and cash equivalents	106,492	65,589
Money market funds | Level 1 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Cash and cash equivalents	106,492	$ 65,589
Certificates of deposits | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	5,461
Certificates of deposits | Level 2 | Fair value, measurements, recurring
Assets, Fair Value Disclosure [Abstract]
Marketable securities	$ 5,461

Cash, cash equivalents, restr_3

Cash, cash equivalents, restricted cash and available-for-sale marketable securities - Cash, cash equivalents, and marketable securities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Amortized Cost
Cash and cash equivalents	$ 112,525	$ 68,802
Total money market funds and marketable securities	339,232	227,583
Debt securities, unrealized loss position	96,000	44,200
Fair Value
Total money market funds and marketable securities	339,326	227,663
Unrealized Gains and Losses
Marketable securities, unrealized gains	158	93
Marketable securities, unrealized losses	(64)	(13)
Money market funds
Amortized Cost
Cash and cash equivalents	106,492	65,589
Fair Value
Cash and cash equivalents, fair value	106,492	65,589
Certificates of deposits
Amortized Cost
Marketable securities, Amortized cost	5,449
Fair Value
Marketable securities, fair value	5,461
Unrealized Gains and Losses
Marketable securities, unrealized gains	12
U.S. Treasury notes
Amortized Cost
Marketable securities, Amortized cost	77,846	102,966
Fair Value
Marketable securities, fair value	77,920	103,044
Unrealized Gains and Losses
Marketable securities, unrealized gains	74	81
Marketable securities, unrealized losses		(3)
U. S. Government agency securities
Amortized Cost
Marketable securities, Amortized cost	24,644	31,068
Fair Value
Marketable securities, fair value	24,655	31,075
Unrealized Gains and Losses
Marketable securities, unrealized gains	13	10
Marketable securities, unrealized losses	(2)	(3)
Corporate bonds
Amortized Cost
Marketable securities, Amortized cost	102,077	23,975
Fair Value
Marketable securities, fair value	102,083	23,970
Unrealized Gains and Losses
Marketable securities, unrealized gains	55	2
Marketable securities, unrealized losses	(49)	(7)
Commercial paper
Amortized Cost
Marketable securities, Amortized cost	22,724	3,985
Fair Value
Marketable securities, fair value	22,715	$ 3,985
Unrealized Gains and Losses
Marketable securities, unrealized gains	4
Marketable securities, unrealized losses	$ (13)

Cash, cash equivalents, restr_4

Cash, cash equivalents, restricted cash and available-for-sale marketable securities - Reconciliation of cash, cash equivalents, and restricted cash (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023	Sep. 30, 2023	Dec. 31, 2022
Cash, cash equivalents, restricted cash, and available-for-sale marketable securities
Cash and cash equivalents	$ 112,525	$ 68,802
Restricted cash included in deposits and other noncurrent assets	2,874	1,593
Total cash, cash equivalents, and restricted cash	$ 115,399	$ 70,395	$ 66,862	$ 100,474

Accrued expenses (Details)

Accrued expenses (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Accrued expenses
Employee compensation costs	$ 5,927	$ 6,614
Research and development costs	3,972	5,225
Accrued goods and services	530	4,229
Professional services	1,013	755
Total	$ 11,442	$ 16,823

Lease obligation (Details)

Lease obligation (Details) $ in Thousands		3 Months Ended			9 Months Ended
	Aug. 11, 2023 USD ($) ft²	Sep. 30, 2024 USD ($)	Jun. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Feb. 01, 2024 USD ($)	Dec. 31, 2023 USD ($)
Operating lease, right-of-use assets		$ 34,523			$ 34,523			$ 13,510
Lease liability		38,368			38,368			17,093
Restricted cash included in deposits and other noncurrent assets		2,874			2,874			$ 1,593
Impairment charge					2,776
Other income from sublease		200			200
Lease expense		$ 1,900		$ 900	$ 8,000	$ 2,700
Weighted average remaining lease term		4 years 10 months 24 days			4 years 10 months 24 days
Weighted average incremental borrowing rate		6.80%			6.80%
75 and 64 Sidney Street
Restricted cash included in deposits and other noncurrent assets		$ 2,900			$ 2,900
64 Sidney Street
Impairment charge			$ 2,800
75 Hayden avenue
Area of additional facility leased | ft²	61,307
Leasehold improvements amount	$ 1,800
Operating lease, right-of-use assets							$ 26,700
Lease liability							$ 26,700

Commitments, contingencies, and

Commitments, contingencies, and other liabilities (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2016 USD ($) multiple	Sep. 30, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2017 USD ($)
Other current liabilities
Lease liability		$ 6,979	$ 3,200
Total other current liabilities		6,979	3,200
Other non-current liabilities
Lease liability		38,368	17,093
Other		1,000	1,001
Total other non-current liabilities		$ 39,368	$ 18,094
Non-profit organization agreement
Other non-current liabilities
Maximum funding	$ 4,000
Funding multiple | multiple	2.6
Milestone payment liability				$ 1,000

Significant Agreements - Narrat

Significant Agreements - Narratives (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration revenue	$ 24,629	$ 4,614	$ 73,723	$ 159,947

Significant Agreements - Novart

Significant Agreements - Novartis Option and License Agreement & Stock Purchase Agreement (Details) $ in Thousands			3 Months Ended		9 Months Ended		12 Months Ended
	Sep. 03, 2024 USD ($)	Dec. 28, 2023 USD ($) shares	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Dec. 31, 2023 Program
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration revenue			$ 24,629	$ 4,614	$ 73,723	$ 159,947
Novartis Pharma, AG | 2023 Novartis Collaboration Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Number of collaboration programs | Program							2
Shares of common stock | shares		2,145,002
Aggregate purchase price		$ 20,000
Amount of premium		$ 700
Novartis Pharma, AG | Option and License Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration revenue			$ 15,000
Upfront payment	$ 15,000
Novartis Pharma, AG | Development, regulatory and commercialization milestone | Option and License Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Aggregate maximum milestone payments to be received from collaborative partner	130,000
Novartis Pharma, AG | Sales milestone | Option and License Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Aggregate maximum milestone payments to be received from collaborative partner	$ 175,000

Significant Agreements - Neuroc

Significant Agreements - Neurocrine Collaboration Agreements (Details) - USD ($) $ in Thousands	1 Months Ended		3 Months Ended				9 Months Ended
	Sep. 30, 2024	Apr. 30, 2024	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration revenue			$ 24,629			$ 4,614	$ 73,723	$ 159,947
2019 Neurocrine Collaborative Agreement | Neurocrine
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Milestone payments					$ 5,000
Allocation of transaction price					5,000
Cumulative catch up of revenue recognized					$ 4,400
2023 Neurocrine Collaborative Agreement | Neurocrine
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration revenue				$ 3,000
Milestone payments	$ 3,000	$ 3,000
Allocation of transaction price	$ 3,000		3,000				$ 3,000
Cumulative catch up of revenue recognized			$ 2,000

Significant Agreements - Neur_2

Significant Agreements - Neurocrine Collaboration receivables and contract liabilities (Details) $ in Thousands	9 Months Ended
	Sep. 30, 2024 USD ($)
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Related party collaboration receivables, beginning balance	$ 3,341
Related party collaboration receivables, ending balance	3,714
2023 Neurocrine Collaborative Agreement and 2019 Neurocrine Collaborative Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Related party collaboration receivables, beginning balance	3,341
Additions	11,895
Deductions	(11,522)
Related party collaboration receivables, ending balance	3,714
Deferred revenue, beginning balance	75,240
Additions	1,592
Deductions	(42,050)
Deferred revenue, ending balance	34,782
Discovery program 3 | Neurocrine | 2023 Neurocrine Collaborative Agreement and 2019 Neurocrine Collaborative Agreement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Additions	1,600
Deductions	$ (42,100)

Stock-based compensation - Comp

Stock-based compensation - Compensation expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	$ 3,434	$ 2,878	$ 11,048	$ 8,108
Employee Stock Option [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	2,373	2,021	7,357	5,587
Restricted stock awards and units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	983	774	3,454	2,339
Employee stock purchase plan awards
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	78	83	237	182
Research and development
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	1,590	919	4,495	2,472
General and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock-based compensation	$ 1,844	$ 1,959	$ 6,553	$ 5,636

Stock-based compensation - Rest

Stock-based compensation - Restricted Stock - (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Restricted stock disclosures
Stock-based compensation.	$ 3,434	$ 2,878	$ 11,048	$ 8,108
Unvested restricted common stock units
Shares
Balance, beginning (in shares)			1,370,897
Granted (in shares)			867,558
Vested (in shares)			(423,362)
Forfeited (in shares)			(198,076)
Balance, ending (in shares)	1,617,017		1,617,017
Weighted Average Grant Date Fair Value Per Share
Balance, beginning (in dollars per share)			$ 6.65
Granted (in dollars per share)			8.15
Vested (in dollars per share)			6.23
Forfeited (in dollars per share)			6.68
Balance, ending (in dollars per share)	$ 7.56		$ 7.56
Restricted stock disclosures
Vesting period			3 years
Stock-based compensation.	$ 983	$ 774	$ 3,454	$ 2,339
Unrecognized stock-based compensation expense	$ 9,100		$ 9,100
Remaining weighted-average remaining vesting period			2 years 1 month 6 days

Stock-based compensation - Stoc

Stock-based compensation - Stock Options - (Details) - USD ($) $ / shares in Units, $ in Thousands	9 Months Ended
	Sep. 30, 2024
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward]
Outstanding, beginning balance (in shares)	7,425,444
Granted (in shares)	2,325,226
Exercised (in shares)	(86,100)
Cancelled or forfeited (in shares)	(793,242)
Outstanding, ending balance (in shares)	8,871,328
Exercisable (in shares)	4,604,321
Weighted Average Exercise Price
Outstanding (in dollars per share)	$ 8.52
Granted (in dollars per share)	7.79
Exercised (in dollars per share)	4.11
Cancelled or forfeited (in dollars per share)	11.69
Outstanding (in dollars per share)	8.08
Exercisable (in dollars per share)	$ 8.51
Remaining Contractual Life
Outstanding	7 years 6 months
Exercisable	6 years 4 months 24 days
Aggregate Intrinsic Value
Outstanding	$ 3,756
Exercisable	2,777
Employee Stock Option [Member]
Options disclosures
Unrecognized stock-based compensation expense	$ 20,100
Remaining weighted-average remaining vesting period	2 years 8 months 12 days

Net (loss) income per share (De

Net (loss) income per share (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share
Antidilutive securities	10,488,345	9,080,820	10,488,345	7,432,212
Unvested restricted common stock awards
Antidilutive Securities Excluded from Computation of Earnings Per Share
Antidilutive securities		22,500		22,500
Unvested restricted common stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share
Antidilutive securities	1,617,017	1,312,727	1,617,017	714,164
Employee Stock Option [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share
Antidilutive securities	8,871,328	7,745,593	8,871,328	6,695,548

Net (loss) income per share - B

Net (loss) income per share - Basic and diluted weighted average shares outstanding (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Numerator:
Net (loss) income	$ (9,044)	$ (25,901)	$ (30,515)	$ 75,935
Denominator for basic net (loss) income per share:
Weighted average shares outstanding-basic	57,851,110	43,864,838	57,564,413	40,962,116
Denominator for diluted net (loss) income per share:
Weighted average shares outstanding-basic	57,851,110	43,864,838	57,564,413	40,962,116
Common stock options and restricted stock units				1,648,608
Weighted average shares outstanding-diluted	57,851,110	43,864,838	57,564,413	42,610,724
Net (loss) income per share, basic	$ (0.16)	$ (0.59)	$ (0.53)	$ 1.85
Net (loss) income per share, diluted	$ (0.16)	$ (0.59)	$ (0.53)	$ 1.78

Underwritten public offering (D

Underwritten public offering (Details) - USD ($) $ / shares in Units, $ in Millions	Jan. 09, 2024	Sep. 30, 2024	Jan. 04, 2024	Dec. 31, 2023
Underwritten public offering
Par value of common stock (in dollars per share)		$ 0.001		$ 0.001
Underwriting Agreement
Underwritten public offering
Issuance of common stock, shares	7,777,778
Par value of common stock (in dollars per share)			$ 0.001
Offering price for common stock			8.46
Net proceeds received	$ 93.5
Underwriting Agreement | Pre-Funded Warrants
Underwritten public offering
Number of warrants issued to purchase common stock	3,333,333
Price per warrant			$ 8.459
Issuance of Pre-Funded Warrants	3,333,333

Related party transactions (Det

Related party transactions (Details) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023	Jun. 30, 2023
Related party transactions
Related party collaboration receivable	$ 3,714,000		$ 3,714,000		$ 3,341,000
Dr. Sah | Related Party
Related party transactions
Total amount of services received	150,000	$ 157,500	450,000	$ 541,300
Advisory fee amount per month						$ 50,000
Neurocrine Collaborative Agreement 2019
Related party transactions
Related party collaboration receivable	400,000		400,000
Neurocrine Collaborative Agreement 2023
Related party transactions
Related party collaboration receivable	$ 3,300,000		$ 3,300,000