Edgewise Therapeutics (EWTX) 10-K 3 Mar 25 2024 FY Annual report Financial data

DOCUMENT AND ENTITY INFORMATION

DOCUMENT AND ENTITY INFORMATION - USD ($) $ in Billions	12 Months Ended
	Dec. 31, 2024	Feb. 24, 2025	Jun. 30, 2024
DOCUMENT AND ENTITY INFORMATION
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2024
Document Transition Report	false
Entity File Number	001-40236
Entity Registrant Name	Edgewise Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	82-1725586
Entity Address, Address Line One	1715 38th St.
Entity Address State Or Province	CO
Entity Address, City or Town	Boulder
Entity Address, Postal Zip Code	80301
City Area Code	720
Local Phone Number	262-7002
Title of 12(b) Security	Common stock, par value $0.0001 per share
Trading Symbol	EWTX
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	true
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		95,205,283
Entity Public Float			$ 1.4
Auditor Name	KPMG LLP
Auditor Firm ID	185
Auditor Location	Denver, Colorado
Entity Central Index Key	0001710072
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	FY
Amendment Flag	false
Documents Incorporated by Reference	Information required by Part III of this Form 10-K is incorporated by reference to the registrant’s proxy statement for the 2025 annual meeting of stockholders, which proxy statement will be filed with the Securities and Exchange Commission within 120 days after the end of the fiscal year covered by this Form 10-K.

BALANCE SHEETS

BALANCE SHEETS - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Current assets
Cash and cash equivalents	$ 41,666	$ 86,097
Marketable securities, available for sale	428,504	232,296
Prepaid expenses and other assets	5,313	8,604
Total current assets	475,483	326,997
Property and equipment, net	9,503	10,443
Operating lease right-of-use asset	1,569	2,247
Other non-current assets	262	348
Total assets	486,817	340,035
Current liabilities
Accounts payable	5,579	4,025
Accrued compensation	10,056	5,695
Accrued other expenses	7,229	6,071
Operating lease liability, current portion	996	980
Total current liabilities	23,860	16,771
Operating lease liability, net of current portion	3,741	4,434
Total liabilities	27,601	21,205
Commitments and contingencies (see note 5)
Stockholders' equity:
Preferred stock, $.0001 par value per share; 200,000,000 shares authorized and no shares issued or outstanding as of December 31, 2024 and December 31, 2023
Common stock, $.0001 par value per share; 1,000,000,000 shares authorized as of December 31, 2024 and December 31, 2023; 94,838,466 shares and 70,453,342 shares issued and outstanding as of December 31, 2024 and December 31, 2023, respectively	9	7
Additional paid-in capital	837,363	563,487
Accumulated other comprehensive income	420	99
Accumulated deficit	(378,576)	(244,763)
Total stockholders' equity	459,216	318,830
Total liabilities and stockholders' equity	$ 486,817	$ 340,035

BALANCE SHEETS (Parenthetical)

BALANCE SHEETS (Parenthetical) - $ / shares	Dec. 31, 2024	Dec. 31, 2023
BALANCE SHEETS
Preferred stock, par value	$ 0.0001	$ 0.0001
Preferred stock, shares authorized (in shares)	200,000,000	200,000,000
Preferred stock, shares issued	0	0
Preferred stock, shares outstanding	0	0
Common stock, par value	$ 0.0001	$ 0.0001
Common stock, shares authorized	1,000,000,000	1,000,000,000
Common stock, shares issued	94,838,466	70,453,342
Common stock, shares outstanding	94,838,466	70,453,342

STATEMENTS OF OPERATIONS AND CO

STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Operating expenses
Research and development	$ 126,966	$ 90,905
General and administrative	31,866	23,452
Total operating expenses	158,832	114,357
Loss from operations	(158,832)	(114,357)
Other income
Interest income	25,019	14,194
Total other income	25,019	14,194
Net loss	(133,813)	(100,163)
Other comprehensive income (loss):
Unrealized gain on available-for-sale securities	321	1,454
Total comprehensive loss	$ (133,492)	$ (98,709)
Net loss per share, basic (in dollars per share)	$ (1.45)	$ (1.57)
Net loss per share, diluted (in dollars per share)	$ (1.45)	$ (1.57)
Weighted-average shares outstanding, basic	92,414,626	63,723,600
Weighted-average shares outstanding, diluted	92,414,626	63,723,600

STATEMENTS OF STOCKHOLDERS' EQU

STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Common Stock	Additional Paid-in Capital	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit	Total
Balance, Beginning at Dec. 31, 2022	$ 6	$ 492,665	$ (1,355)	$ (144,600)	$ 346,716
Balance, Beginning (in shares) at Dec. 31, 2022	63,257,376
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net of offering costs	$ 1	52,549			52,550
Issuance of common stock, net of offering costs (in shares)	6,928,044
Exercise of stock options and vesting of restricted stock units		117			117
Exercise of stock options and vesting of restricted stock units (in shares)	172,663
Purchase of common stock under employee stock purchase plan		596			596
Purchase of common stock under employee stock purchase plan (in shares)	95,259
Stock-based compensation		17,560			17,560
Other comprehensive income			1,454		1,454
Net loss				(100,163)	(100,163)
Balance, Ending at Dec. 31, 2023	$ 7	563,487	99	(244,763)	$ 318,830
Balance, Ending (in shares) at Dec. 31, 2023	70,453,342				70,453,342
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock, net of offering costs	$ 2	238,797			$ 238,799
Issuance of common stock, net of offering costs (in shares)	22,450,206
Exercise of stock options and vesting of restricted stock units		9,480			9,480
Exercise of stock options and vesting of restricted stock units (in shares)	1,787,832
Purchase of common stock under employee stock purchase plan		888			888
Purchase of common stock under employee stock purchase plan (in shares)	147,086
Stock-based compensation		24,711			24,711
Other comprehensive income			321		321
Net loss				(133,813)	(133,813)
Balance, Ending at Dec. 31, 2024	$ 9	$ 837,363	$ 420	$ (378,576)	$ 459,216
Balance, Ending (in shares) at Dec. 31, 2024	94,838,466				94,838,466

STATEMENTS OF CASH FLOWS

STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Cash flows from operating activities
Net loss	$ (133,813)	$ (100,163)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	2,069	1,551
Stock-based compensation	24,712	17,560
Accretion of discount on marketable securities, net	(12,545)	(9,517)
Amortization of right-of-use asset	221	181
Changes in assets and liabilities:
Prepaid expenses and other assets	3,292	(3,553)
Accounts payable	1,737	(92)
Accrued compensation	4,361	1,663
Accrued other expenses and other liabilities	1,158	526
Lease liability	(220)	(104)
Net cash used in operating activities	(109,028)	(91,948)
Cash flows from investing activities
Purchases of marketable securities	(477,055)	(255,866)
Sales of marketable securities	15,794	20,118
Maturities of marketable securities	277,917	344,379
Purchases of property and equipment	(1,312)	(5,745)
Net cash (used in) provided by investing activities	(184,656)	102,886
Cash flows from financing activities
Proceeds from issuance of common stock, net of underwriting discounts and commissions and offering costs	239,147	52,618
Exercise of stock options	9,480	117
Payment of deferred offering costs	(262)	(165)
Proceeds from Employee Stock Purchase Plan	888	596
Net cash provided by financing activities	249,253	53,166
Net change in cash and cash equivalents	(44,431)	64,104
Cash and cash equivalents at beginning of period	86,097	21,993
Cash and cash equivalents at end of period	41,666	86,097
Supplemental disclosures of non-cash investing and financing activities:
Right-of-use asset obtained in exchange for new operating lease liability, net of tenant improvement receivable		1,110
Property and equipment purchases included in accounts payable and accrued other expenses	$ 44	$ 227

DESCRIPTION OF BUSINESS

DESCRIPTION OF BUSINESS	12 Months Ended
	Dec. 31, 2024
DESCRIPTION OF BUSINESS
DESCRIPTION OF BUSINESS	NOTE 1 DESCRIPTION OF BUSINESS Organization and Description of Business Edgewise Therapeutics, Inc. (the Company) was incorporated as a Delaware corporation in May 2017, and it is headquartered in Boulder, Colorado. The Company is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe muscle diseases for which there is significant unmet medical need. The Company’s lead product candidates are sevasemten and EDG-7500. Sevasemten is an orally administered small molecule designed to address the root cause of dystrophinopathies including Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker). The Company is currently studying sevasemten in Phase 2 trials, which are being held in the U.S. and Israel and in certain countries in Europe and Australasia. The Company is enrolling a multipart Phase 2 trial with EDG-7500, for the potential treatment of hypertrophic cardiomyopathy (HCM). The Company is using its proprietary drug discovery platform to develop a pipeline of precision medicine product candidates that target key muscle proteins and modulators to address a broad array of serious muscle disorders. Risks and Uncertainties The board of directors of the Company discusses with management macroeconomic and geopolitical developments, including inflation, instability in the banking and financial services sector, tightening of the credit markets, the impact of the change in the U.S. presidential administration, international conflicts, public health pandemics, cybersecurity and sanctions so that the Company can be prepared to react to new developments as they arise. The board of directors and the management of the Company are carefully monitoring these developments and the resulting economic impact on its financial condition and results of operations. Liquidity and Capital Resources The Company has an accumulated deficit of $378.6 million and, cash, cash equivalents and marketable securities of $470.2 million as of December 31, 2024. The Company’s ability to fund ongoing operations is highly dependent upon raising additional capital through the issuance of equity securities and issuing debt or other financing vehicles. On June 16, 2023, the Company entered into a Sales Agreement (Sales Agreement) with BofA Securities, Inc. (BofA Securities) under which the Company could offer and sell shares of common stock, having aggregate sales proceeds of up to $125,000,000 from time to time, through an “at the market offering” program (ATM Program) under which BofA Securities acted as sales agent. Effective January 19, 2024, the Company suspended and terminated the prospectus related to the Company’s common stock issuable pursuant to the terms of the Sales Agreement (the ATM Prospectus). As of the date of the suspension of the ATM Prospectus, the Company had sold 7,560,068 shares of our common stock at a weighted average price of $7.93 per share. The gross proceeds were $59.9 million, and the net proceeds were $59.4 million after deducting underwriting discounts and commissions of $0.2 million and offering expenses of $0.3 million. On January 23, 2024, the Company closed an underwritten registered direct offering of 21,818,182 shares of common stock at a public offering price of $11.00 per share (the January 2024 Offering). The aggregate gross proceeds from the January 2024 Offering were $240.0 million, and the net proceeds were $231.9 million after deducting underwriting discounts and commissions of $7.5 million and offering expenses of $0.6 million. On May 10, 2024, we filed an automatic shelf registration statement on Form S-3ASR that allows us to undertake various equity and debt offerings. Additionally, the Company filed a prospectus supplement to the shelf registration statement and entered into a sales agreement with Leerink Partners LLC (Leerink Sales Agreement) under which the Company may offer and sell shares of common stock, having aggregate sales proceeds of up to $175,000,000 from time to time, through an “at the market offering” program (Leerink ATM) under which Leerink Partners LLC will act as sales agent. The Company has not yet offered or sold any shares of common stock related to the Leerink ATM. The Company’s ability to secure capital is dependent upon success in developing its technology and product candidates. The Company cannot provide assurance that additional capital will be available on acceptable terms, if at all. The issuance of additional equity or debt securities will likely result in substantial dilution to the Company’s stockholders. Should additional capital not be available to the Company in the near term, or not be available on acceptable terms, the Company may be unable to realize value from the Company’s assets or discharge liabilities in the normal course of business, which may, among other alternatives, cause the Company to delay, substantially reduce, or discontinue operational activities to conserve cash balances, which could have a material adverse effect on the Company’s ability to achieve its intended business objectives. The accompanying financial statements have been prepared assuming that the Company will continue as a going concern, which contemplates the realization of assets and the settlement of liabilities and commitments in the normal course of business. The financial statements do not reflect any adjustments relating to the recoverability and reclassification of assets and liabilities that might be necessary if the Company is unable to continue as a going concern. The Company believes that the $470.2 million of cash, cash equivalents and marketable securities on hand as of December 31, 2024 will be sufficient to fund its operations in the normal course of business and meet its liquidity needs through at least the next 12 months from the issuance of these financial statements.

SUMMARY OF SIGNIFICANT ACCOUNTI

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES	12 Months Ended
	Dec. 31, 2024
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES	NOTE 2 SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Basis of Presentation The accompanying financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (U.S. GAAP). Segment Information The Company is organized as a single operating and reportable segment, focused on the discovery, development, and manufacture of drug products for the treatment of severe muscle diseases; segment information is presented in Note 11. All equipment and other fixed assets are physically located in the United States. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Actual results could differ from those estimates. Cash Equivalents The Company considers all liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents as of December 31, 2024 and 2023 primarily consist of money market funds and cash. Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentration of credit risk consist primarily of cash, cash equivalents and marketable securities. Periodically, the Company may maintain deposits in financial institutions in excess of government insured limits. The Company believes that it is not exposed to significant credit risk as its deposits are held at financial institutions that management believes to be of high credit quality. The Company has not experienced any losses on deposits since inception. The Company regularly invests excess cash with major financial institutions in money market funds, corporate debt securities, and commercial paper, all of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from our holdings of these financial instruments is mitigated based on the fact that many of these securities are of high credit rating. Deferred Offering Costs The Company capitalizes certain legal, professional, accounting and other third-party fees that are directly associated with in-process equity issuances as deferred offering costs until such equity issuances are consummated. After consummation of the equity issuance, these costs are recorded as a reduction in the capitalized amount associated with the equity issuance. Should the equity issuance be abandoned, the deferred offering costs are expensed immediately as a charge to operating expenses in the statement of operations. Deferred offering costs as of December 31, 2024 and December 31, 2023 were $0.3 million. Such costs are classified in other non-current assets in the accompanying balance sheets. Property and Equipment Property and equipment is stated at cost less accumulated depreciation and amortization. Depreciation of property and equipment is computed using the straight-line method over the estimated useful life of the related asset, which is generally three Leases The Company accounts for its leases under Accounting Standards Codification (ASC) Topic 842, Leases Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. For all asset classes of its leases, the Company has elected to account for the lease and non-lease components together for existing classes of underlying asset. Costs determined to be variable and not based on an index or rate are not included in the measurement of the lease liability. Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or circumstances indicate that the carrying value of such assets may not be fully recoverable. Impairment is evaluated based on the sum of undiscounted estimated future cash flows expected to result from use of the related asset compared to its carrying value. If impairment is recognized, the carrying value of the impaired asset is reduced to its fair value. There were no impairment charges or long-lived assets disposed of during the years ended December 31, 2024 and 2023. Income Taxes Deferred income taxes are provided on temporary differences between financial statement and income tax reporting. Temporary differences are differences between the amounts of assets and liabilities reported for financial statement purposes and their tax bases. Deferred tax assets are recognized for temporary differences that will be deductible in future years’ tax returns and for operating loss and tax credit carryforwards. Deferred tax assets are reduced by a valuation allowance if such deferred tax assets are deemed more likely than not that some or all of the deferred tax assets will not be realized. Historically, the Company has not recognized these potential benefits in its financial statements and has fully reserved for such net deferred tax assets, as it believes it is more likely than not that the full benefit of these net deferred tax assets will not be realized. Deferred tax liabilities are recognized for temporary differences that will be taxable in future years. The Company evaluated its tax positions and determined it has no uncertain tax positions as of December 31, 2024. Fair Value of Financial Instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. The Financial Accounting Standards Board (FASB) ASC Topic 820, Fair Value Measurements and Disclosures Level 1—quoted prices in active markets for identical assets and liabilities. Level 2—other significant observable inputs (including quoted prices for similar assets and liabilities, interest rates, credit risk, etc.). Level 3—significant unobservable inputs (including the Company’s own assumptions in determining the fair value of assets and liabilities). Marketable Securities, Available For Sale All marketable securities have been classified as “available-for-sale” and are carried at fair value, based upon quoted market prices. The Company considers its available-for-sale portfolio as available for use in current operations. Accordingly, the Company classifies its investments as short-term marketable securities, even though the stated maturity date may be one year or more beyond the current balance sheet date. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive income (loss) and reported as a separate component of stockholders’ equity until realized. Interest income, realized gains and losses, and declines in value judged to be other than temporary, if any, on available-for-sale securities are included in other income. 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. In accordance with the Company’s investment policy, management invests in money market funds, corporate debt securities, commercial paper, asset-backed securities and government securities. The Company has not experienced any realized losses on its deposits of cash, cash equivalents, and marketable securities since inception. The following tables summarize the Company’s financial assets measured at fair value on a recurring basis by level within the fair value hierarchy (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2024 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ Hierarchy Cost Basis Gains Losses Value Cash equivalents: ​ ​ ​ ​ Money market funds Level 1 ​ $ 41,474 ​ $ — ​ $ — ​ $ 41,474 Marketable securities, available for sale: ​ ​ ​ ​ ​ ​ Asset-backed securities Level 2 ​ 50,262 ​ $ 69 ​ $ — ​ 50,331 Corporate debt securities Level 2 ​ 193,769 ​ $ 179 ​ $ (81) ​ 193,867 Commercial paper Level 2 ​ 10,432 ​ $ 3 ​ $ — ​ 10,435 U.S. government treasury and agency securities ​ Level 2 ​ 173,621 ​ $ 279 ​ $ (29) ​ 173,871 Total ​ $ 469,558 ​ $ 530 ​ $ (110) ​ $ 469,978 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2023 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ ​ Hierarchy Cost Basis Gains Losses Value Cash equivalents: ​ ​ ​ ​ Money market funds Level 1 ​ $ 85,897 ​ $ — ​ $ — ​ $ 85,897 Marketable securities, available for sale: ​ ​ ​ ​ Asset-backed securities Level 2 ​ 10,228 ​ 12 ​ (2) ​ 10,238 Corporate debt securities Level 2 ​ 82,514 ​ 66 ​ (113) ​ 82,467 Commercial paper ​ Level 2 ​ ​ 19,457 ​ ​ 13 ​ ​ (8) ​ ​ 19,462 U.S. government treasury and agency securities ​ Level 2 ​ ​ 116,579 ​ ​ 151 ​ ​ (26) ​ ​ 116,704 Supranational and sovereign government securities Level 2 ​ 3,419 ​ 6 ​ — ​ 3,425 Total ​ $ 318,094 ​ $ 248 ​ $ (149) ​ $ 318,193 ​ The Company’s money market funds are classified as Level 1 because they are valued using quoted market prices. Investments in asset-backed securities, corporate debt securities, commercial paper and U.S. government treasury and agency securities, and supranational and sovereign government securities have been classified as Level 2 as they are valued using quoted prices in less active markets or other directly or indirectly observable inputs. Fair values of asset-backed securities, corporate debt securities, commercial paper, U.S. government treasury and agency securities, and supranational and sovereign government securities were derived based on input of market prices from multiple sources at each reporting period. With regard to commercial paper, all of the securities had high credit ratings and one year or less to maturity; therefore, fair value was derived from accretion of purchase price to face value over the term of maturity or quoted market prices for similar instruments if available. There were no transfers of financial assets between Level 1, Level 2, or Level 3, during the periods presented. As of December 31, 2024, remaining contractual maturities of $394.4 million of marketable securities were less than one year 1 The Company periodically reviews its portfolio of debt securities to determine if any investment is impaired due to credit loss or other potential valuation concerns. For debt securities where the fair value of the investment is less than the amortized cost basis, the Company has assessed at the individual security level for various quantitative factors including, but not limited to, the nature of the investments, changes in credit ratings, interest rate fluctuations, industry analyst reports, and the severity of impairment. Unrealized losses on marketable securities at December 31, 2024 were primarily due to changes in interest rates, including market credit spreads, and not due to increased credit risks associated with specific securities. Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in equity during a period from transactions and other events and/or circumstances from non-owner sources. The Company’s only element of other comprehensive income (loss) was net unrealized gain (loss) on marketable securities. Stock-Based Compensation In accordance with ASC Topic 718, Compensation—Stock Compensation Research and Development Expenses and Accrued Research and Development Expenses Expenditures made for research and development are charged to expense as incurred. External costs consist primarily of payments to contract research organizations (CROs), contract development and manufacturing organizations (CDMOs), sample acquisition costs and laboratory supplies purchased in connection with the Company’s discovery and preclinical activities, and process development and clinical development activities. Internal costs consist primarily of employee-related costs, facilities, depreciation and costs related to compliance with regulatory requirements. Non-refundable advance payments for goods and services that will be used in future research and development activities are capitalized and recorded as an expense in the period that the Company receives the goods or when services are performed. The Company records expenses related to external research and development services based on its estimates of the services received and efforts expended pursuant to quotes and contracts with multiple CROs and CDMOs that supply, conduct and manage preclinical studies and clinical trials on its behalf. The financial terms of these contracts vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided under such contracts. 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. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual or the amount of prepaid expenses accordingly. Recently Adopted Accounting Pronouncements In June 2016, the FASB issued Accounting Standards Update (ASU) 2016-13, Financial Instruments - Credit Losses: Measurement of Credit Losses on Financial Statements ​ In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures Accounting Standards Not Yet Adopted In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures In November 2024, the FASB issued ASU 2024-03, Income Statement–Reporting Comprehensive Income–Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses

PREFERRED STOCK AND COMMON STOC

PREFERRED STOCK AND COMMON STOCK	12 Months Ended
	Dec. 31, 2024
PREFERRED STOCK AND COMMON STOCK
PREFERRED STOCK AND COMMON STOCK	NOTE 3 PREFERRED STOCK AND COMMON STOCK The Company is authorized to issue two classes of stock designated as common stock and preferred stock. As of December 31, 2024 the total number of shares authorized was 1,200,000,000. The total number of shares of common stock authorized was 1,000,000,000. The total number of shares of preferred stock authorized was 200,000,000. All shares of the Company’s capital stock have a par Common stockholders are entitled to dividends if and when declared by the board of directors of the Company and after any convertible preferred share dividends are fully paid. The holder of each share of common stock is entitled to one vote.

STOCK-BASED COMPENSATION AWARDS

STOCK-BASED COMPENSATION AWARDS	12 Months Ended
	Dec. 31, 2024
STOCK-BASED COMPENSATION AWARDS
STOCK-BASED COMPENSATION AWARDS	NOTE 4 Equity Incentive Plans In March 2021, the Company’s board of directors adopted, and its stockholders approved, the Company’s 2021 Equity Incentive Plan (the 2021 Plan), which became effective in March 2021 in connection with the IPO. Upon adoption of the 2021 Plan, the Company restricted the grant of future equity awards under its 2017 Equity Incentive Plan, as amended and restated (the 2017 Plan). The 2021 Plan provides for the grant of incentive stock options, within the meaning of Section 422 of the Internal Revenue Code, to the Company’s employees and any of its parent and subsidiary corporations’ employees, and for the grant of nonstatutory stock options, restricted stock, restricted stock units, stock appreciation rights, performance units, and performance shares to its employees, directors, and consultants and its subsidiary corporations’ employees and consultants. The vesting of stock options is stated in each individual grant agreement, which is generally four years. Options granted expire 10 years after the date of grant. A total of 5,040,000 shares of the Company’s common stock were initially reserved for issuance pursuant to the 2021 Plan. The 2021 Plan share reserve increases by the number of shares under the 2017 Plan that are repurchased, forfeited, expired or cancelled after the effective date of the 2021 Plan up to the limit under the 2021 Plan. The number of shares available for issuance under the 2021 Plan increases annually on the first day of each fiscal year beginning with the Company’s 2022 fiscal year, equal to the least of (1) 5,040,000 shares, (2) five percent (5%) of the outstanding shares of its common stock as of the last day of the immediately preceding fiscal year; or (3) such other amount as the Company’s board of directors may determine. As of December 31, 2024, there were 1,453,866 shares available for future issuance under the 2021 Plan. Stock Options Stock option activity for the year ended December 31, 2024 is as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Average ​ ​ ​ ​ Weighted ​ ​ ​ ​ Remaining ​ ​ ​ ​ Average ​ Total Intrinsic ​ Contractual Life ​ Options Exercise Price Value (000's) (Years) Outstanding as of December 31, 2023 14,491,613 $ 7.43 $ 65,801 ​ 8.0 Granted 2,817,497 ​ $ 17.67 ​ ​ ​ ​ ​ Exercised (1,621,074) ​ $ 6.00 ​ ​ ​ ​ ​ Cancelled ​ (271,275) ​ $ 10.41 ​ ​ ​ ​ ​ Outstanding as of December 31, 2024 ​ 15,416,761 ​ $ 9.40 ​ $ 267,397 ​ 7.5 Options exercisable as of December 31, 2024 ​ 8,643,539 ​ $ 7.13 ​ $ 169,659 ​ 6.6 ​ The vesting of stock options is stated in each individual grant agreement, which is generally four years. As of December 31, 2024, there was unrecognized stock-based compensation cost of $56.1 million, which is expected to be recognized over a weighted-average term of 2.7 years. The aggregate intrinsic value of options exercised during the years ended December 31, 2024 and 2023 was $30.3 million and $1.0 million, respectively. For options granted during the years ended December 31, 2024 and 2023, the weighted-average grant date fair value was $12.73 and $5.25 per share, respectively. In previous years, there were additional options issued outside of this plan as discussed under Founder Stock Options. Inducement Equity Incentive Plan Effective August 10, 2024, the Company’s board of directors adopted the Company’s 2024 Inducement Equity Incentive Plan (Inducement Plan) and, subject to the adjustment provisions of the Inducement Plan, reserved 2,000,000 shares of the Company’s common stock for issuance pursuant to equity awards granted under the Inducement Plan. The Inducement Plan was adopted without stockholder approval pursuant to the applicable The Nasdaq Stock Market LLC’s (Nasdaq) Listing Rules. The Inducement Plan provides for the grant of equity-based awards, including nonstatutory stock options, stock appreciation rights, restricted stock, restricted stock units, and performance awards, and its terms are substantially similar to the 2021 Plan, including with respect to treatment of equity awards in the event of a “merger” or “change in control” as defined under the Inducement Plan, but with such other terms and conditions intended to comply with the Nasdaq inducement award exception or to comply with the Nasdaq acquisition and merger exception. In accordance with the Nasdaq Listing Rules, awards under the Inducement Plan may only be made to individuals not previously employees or non-employee directors of the Company (or following such individuals’ bona fide period of non-employment with the Company), as an inducement material to the individuals’ entry into employment with the Company, or, to the extent permitted by the Nasdaq Listing Rules, in connection with a merger or acquisition. As of December 31, 2024, there were 2,000,000 shares available for future issuance under the Inducement Plan. Founder Stock Options On September 19, 2017, the Company granted one of its founders the option to purchase 1,795,880 shares of the Company’s common stock at an exercise price of $0.18 per share which vest monthly over a four-year period that expires 15 years after the date of grant. This grant is separate from the Company’s equity incentive plans discussed above. As of December 31, 2024, 1,356,780 options were both outstanding and exercisable Fair Value Assumptions The fair value of option grants is estimated on the date of grant using the Black-Scholes option-pricing model, which requires the use of the following assumptions: ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 Expected term (Years) ​ 5.10 - 6.85 ​ 5.44 - 6.85 Expected volatility ​ 78.67% - 83.21% ​ 80.68% - 85.68% Risk-free interest rate ​ 3.59% - 4.66% ​ 3.47% - 4.80% Expected dividend rate ​ - ​ - Fair value common stock ​ $16.33 - $33.57 ​ $5.73 - $10.29 ​ The expected term is based on the “simplified method” described in the U.S. Securities and Exchange Commission’s Staff Accounting Bulletin Topic 14 which is determined as the midpoint between the vesting date and the contractual end of the option grant. Stock price volatility was estimated based on the estimated stock price volatility of a peer group of publicly traded companies over a similar term. The risk-free interest rate for periods within the contractual life of the option is based on the U.S. Treasury yield in effect at the time of grant. The dividend yield was zero as the Company has never declared or paid dividends and has no plans to do so in the foreseeable future. ​ ​ ​ Restricted Stock Units A restricted stock unit (RSU) represents the right to receive one share of common stock upon vesting of the RSU. The fair value of each RSU is based on the closing price of the Company’s common stock on the date of grant and vest over 3 RSU activity for the year ended December 31, 2024 is as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted Average ​ ​ ​ ​ Grant Date ​ RSUs Fair Value Issued and unvested as of December 31, 2023 177,871 $ 7.98 Granted 557,027 ​ $ 17.05 Vested (84,855) ​ $ 7.98 Cancelled ​ (10,527) ​ $ 10.03 Issued and unvested as of December 31, 2024 ​ 639,516 ​ $ 15.85 ​ As of December 31, 2024, there was unrecognized stock-based compensation cost of $8.7 million, which is expected to be recognized over a weighted-average term of 3.5 years. The total fair value of RSUs vested during each of the years ended December 31, 2024 and 2023 was $0.7 million. 2021 Employee Stock Purchase Plan The 2021 Employee Stock Purchase Plan (2021 ESPP) enables eligible employees of the Company to purchase shares of common stock at a discount. A total of 504,000 shares of the Company’s common stock were initially reserved for issuance pursuant to the 2021 ESPP. The number of shares available for issuance under the 2021 ESPP increases annually on the first day of each fiscal year beginning with the Company’s 2022 fiscal year, equal to the least of (1) 1,008,000 shares, (2) one percent (1%) of the outstanding shares of common stock as of the last day of the immediately preceding fiscal year; or (3) such other amount as the Company’s board of directors may determine. As of December 31, 2024, the Company has reserved for issuance 2,027,407 shares of common stock pursuant to the 2021 ESPP. The 2021 ESPP provides for two offering periods of up to twelve months’ duration with purchase periods commencing on the first trading day on or after either May 15 and November 15, and terminating the last trading day on or before either May 15 or November 15 of the same year, and May 15 of the following year, respectively. Contributions under the 2021 ESPP are limited to 15% of an employee’s eligible compensation, IRS limitations, and a maximum of 6,000 shares during each offering period. 2021 ESPP participants will purchase shares of common stock at a price per share equal to 85% of the lesser of (1) the fair market value per share of the common stock on the first trading day of the offering period or (2) the fair market value of the common stock on the purchase date. Additionally, during the years ended December 31, 2024 and 2023, a total of 147,086 and 95,259 shares of common stock were issued under the ESPP at a weighted-average per share price of $6.03 and $6.26, respectively. Total stock-based compensation expense related to all equity plans, including Founder Stock Options was allocated as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 Research and development $ 13,956 $ 10,029 General and administrative ​ 10,755 ​ 7,531 Total stock-based compensation expense ​ $ 24,711 ​ $ 17,560 ​

COMMITMENTS AND CONTINGENCIES

COMMITMENTS AND CONTINGENCIES	12 Months Ended
	Dec. 31, 2024
COMMITMENTS AND CONTINGENCIES.
COMMITMENTS AND CONTINGENCIES	NOTE 5 Lease Agreements In January 2022, the Company entered into a lease agreement for approximately 18,614 square feet of office and laboratory space in Boulder, Colorado (the New Boulder Lease) with aggregate base rent payments of approximately $3.3 million over the initial 8.2-year term of the lease. Further, the Company provided a standby letter of credit (LOC) of $0.8 million during the term of the lease as collateral for the Company’s obligations under the lease. Provided there has been no event of default by the Company during the initial 36-month In February 2023, the New Boulder Lease was modified to occupy an additional 9,624 square feet of office space (the Expansion Space) with aggregate payments of approximately $1.5 million over the initial 7.3 year term of the lease. The Expansion space includes an improvement allowance in the amount of $0.5 million to be fully reimbursed by the lessor. As of December 31, 2024, $0.5 million was received by the lessor under the allowance associated with the expansion space. Under the New Boulder Lease and the Expansion Space (collectively, the Lease), the Company has the option to extend the Lease for two additional terms of five years each. The Company is obligated to pay the lessor an amount not to exceed 5% of the net rents from the property for operating costs. Such amounts are not included in the measurement of the lease liabilities and are recognized as variable lease expense when they are incurred. Variable lease expense was $0.5 million and $0.3 million for the years ended December 31, 2024 and 2023, respectively. The Lease is classified as an operating lease. The Company recorded lease liabilities and ROU lease assets for the Lease based on the present value of lease payments over the expected lease term, discounted using the Company’s incremental borrowing rate. The option to extend the Lease was not recognized as part of the Company’s lease liabilities and ROU lease assets, as such extensions are not reasonably certain to occur. As of December 31, 2024, the weighted-average remaining lease term and the weighted-average discount rate for the Lease was 5.3 years and 6.4%, respectively. Rent expense under the Lease was $0.5 million for each of the years ended December 31, 2024 and 2023. Future minimum lease payments under the Lease as of December 31, 2024 are as follows (in thousands): ​ ​ ​ ​ ​ Year Ending December 31, ​ ​ ​ 2025 $ 1,031 2026 ​ ​ 1,048 2027 ​ 1,066 2028 ​ ​ 1,084 2029 ​ ​ 1,103 Thereafter ​ ​ 278 Total undiscounted future minimum lease payments ​ ​ 5,610 Less: discount ​ ​ (873) Total lease liability ​ $ 4,737 ​ Litigation Liabilities for loss contingencies arising from claims, assessments, litigation, fines, penalties and other sources are recorded when it is probable that a liability has been incurred and the amount can be reasonably estimated. From time to time, the Company may become involved in legal proceedings arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the year ended December 31, 2024 and no material legal proceedings are currently pending or threatened. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners and other parties with respect to certain matters including, but not limited to, losses arising of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company is not aware of any claims under indemnification arrangements, and it has not accrued any liabilities related to such obligations in its financial statements as of December 31, 2024.

INCOME TAXES

INCOME TAXES	12 Months Ended
	Dec. 31, 2024
INCOME TAXES
INCOME TAXES	NOTE 6 INCOME TAXES Since inception, the Company has incurred net taxable losses, and accordingly, no current provision for income taxes has been recorded. The effective income tax rate of the provision for income taxes differs from the federal statutory rate as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ 2024 2023 Federal tax at statutory rate ​ 21.0 % ​ 21.0 % Research and development credits ​ 5.6 % ​ 5.8 % State taxes, net of federal deduction ​ 5.5 % ​ 4.9 % Other ​ 3.4 % ​ 0.1 % Disallowed compensation ​ (4.0) % ​ (1.8) % Change in valuation allowance ​ (31.5) % ​ (30.0) % Effective income tax rate ​ — ​ — ​ ​ Deferred tax assets and liabilities are as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ 2024 2023 Deferred tax assets (liabilities): ​ ​ ​ ​ ​ ​ Capitalized research expenses ​ $ 49,054 ​ $ 25,316 Federal net operating loss carryforward ​ ​ 30,714 ​ ​ 22,971 Federal and state R&D credits ​ ​ 16,854 ​ ​ 8,913 State net operating loss carryforward ​ ​ 6,956 ​ ​ 5,197 Stock-based compensation ​ ​ 4,929 ​ ​ 4,120 Accrued expenses ​ ​ 1,661 ​ ​ 1,499 Lease liability ​ ​ 1,236 ​ ​ 1,411 Orphan Drug Credit ​ ​ 606 ​ ​ 691 Property and equipment ​ ​ 107 ​ ​ 30 Other ​ ​ (15) ​ ​ 53 ROU asset ​ ​ (410) ​ ​ (586) Total net deferred tax asset ​ ​ 111,692 ​ ​ 69,615 Valuation allowance ​ ​ (111,692) ​ ​ (69,615) Net deferred tax asset (liability) ​ $ — ​ $ — ​ For the period ended December 31, 2024, the Company has federal gross net operating loss (NOL) carryforwards of $146.3 million. Of this amount, $1.2 million have a limited carryforward period and will begin to expire in 2037; the remaining $145.1 million have an indefinite carryforward period. The Company also has post-apportioned gross state NOL carryforwards of $155.6 million. Of this amount, $122.9 million have a limited carryforward period and will begin to expire in 2037; the remaining $32.7 million have an indefinite carryforward period. The Company also has $16.6 million of federal research related tax credits carryforwards, which will begin to expire in 2039. The Company also has $0.9 million of state research related tax credits carryforward, of which $0.5 million will begin to expire in 2037; the remaining $0.4 million have an indefinite carryforward period. In accordance with Section 382 and Section 383, utilization of the NOL and tax credit carryforwards may subject to limitations based on prior or future ownership changes. Also, after weighing all available and positive and negative evidence, the Company determined a full valuation allowance was necessary, consistent with prior year. The Company is subject to income tax in multiple jurisdictions, including federal and several states. The Company has federal and state income tax returns that are open to examination from 2021 and 2020 forward, respectively. In addition, the utilization of NOLs and tax credit carryforwards, from periods prior to those previously mentioned may also be audited by the taxing authorities once utilized. As a result, the Company continuously monitors its current and prior filing positions in order to determine if any unrecognized tax positions need to be recorded. The analysis involves considerable judgement and is based on the best information available. For the period ended December 31, 2024, the Company is not aware of any positions which require an uncertain tax position liability. Pursuant to Internal Revenue Code (IRC) Sections 382 and 383, the Company’s ability to use NOL and research tax credit carry forwards to offset future taxable income may be limited if the Company experiences a cumulative change in ownership of more than 50% within a three-year testing period. The Company has not completed an ownership change analysis pursuant to IRC Section 382. If ownership changes within the meaning of IRC Section 382 are identified as having occurred, the amount of NOL and research tax carryforwards available to offset future taxable income and income tax liabilities in future years may be significantly restricted or eliminated. Further, deferred tax assets associated with such NOLs and research tax credits could be significantly reduced upon realization of an ownership change within the meaning of IRC Section 382.

EMPLOYEE BENEFIT PLANS

EMPLOYEE BENEFIT PLANS	12 Months Ended
	Dec. 31, 2024
EMPLOYEE BENEFIT PLANS
EMPLOYEE BENEFIT PLANS	NOTE 7 EMPLOYEE BENEFIT PLANS In 2017, the Company established a qualified 401(k) plan which covers all employees who meet eligibility requirements. The Company’s contribution to the plan, as determined by the Company’s Board of Directors, was discretionary until September 2021 when the Company initiated a match with a maximum amount of 4% of the participant’s compensation. During the years ended December 31, 2024 and 2023, the Company made matching contributions of $1.0 million and $0.6 million, respectively.

NET LOSS PER SHARE

NET LOSS PER SHARE	12 Months Ended
	Dec. 31, 2024
NET LOSS PER SHARE
NET LOSS PER SHARE	NOTE 8 NET LOSS PER SHARE Basic net loss per common share is calculated by dividing the net loss by the weighted-average number of common shares outstanding during the period, without consideration of potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss by the weighted-average number of common shares and potentially dilutive securities outstanding for the period. For purposes of the diluted net loss per share calculation, common stock options and unvested restricted stock units are considered to be potentially dilutive securities. The Company’s participating securities do not have a contractual obligation to share in the Company’s losses. As such, the net loss was attributed entirely to common stockholders. As the Company has reported a net loss for all periods presented, diluted net loss per common share is the same as basic net loss per common share for those periods. The following table sets forth the computation of the basic and diluted net loss per share (in thousands, except share and per share data): ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2024 2023 Numerator ​ ​ ​ ​ ​ ​ Net loss ​ $ (133,813) $ (100,163) Denominator ​ ​ ​ ​ Weighted-average shares outstanding used in computing net loss per share, basic and diluted ​ ​ 92,414,626 ​ ​ 63,723,600 Net loss per share, basic and diluted ​ $ (1.45) ​ $ (1.57) ​ The following weighted average outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2024 2023 Options to purchase common stock ​ ​ 16,340,740 ​ ​ 13,764,188 Unvested restricted stock units ​ ​ 328,984 ​ ​ 207,628 Total ​ ​ 16,669,724 ​ ​ 13,971,816 ​

PROPERTY AND EQUIPMENT

PROPERTY AND EQUIPMENT	12 Months Ended
	Dec. 31, 2024
PROPERTY AND EQUIPMENT
PROPERTY AND EQUIPMENT	NOTE 9 PROPERTY AND EQUIPMENT Property and equipment consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ As of December 31, ​ 2024 2023 Leasehold improvements ​ $ 9,646 ​ $ 8,728 Laboratory equipment ​ ​ 3,735 ​ ​ 3,473 Computers and software ​ ​ 296 ​ ​ 281 Furniture and fixtures ​ ​ 511 ​ ​ 497 Construction in process ​ ​ — ​ ​ 80 Property and equipment, at cost ​ ​ 14,188 ​ ​ 13,059 Less: accumulated depreciation ​ ​ (4,685) ​ ​ (2,616) Property and equipment, net ​ $ 9,503 ​ $ 10,443 ​ Depreciation expense was $2.1 million and $1.6 million for the years ended December 31, 2024 and 2023, respectively.

ACCRUED OTHER EXPENSES

ACCRUED OTHER EXPENSES	12 Months Ended
	Dec. 31, 2024
ACCRUED OTHER EXPENSES
ACCRUED OTHER EXPENSES	NOTE 10 ACCRUED OTHER EXPENSES Accrued other expenses consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ As of December 31, ​ 2024 2023 Accrued research and development costs ​ $ 6,488 ​ $ 5,672 Accrued other ​ ​ 741 ​ ​ 399 Total accrued other expenses ​ $ 7,229 ​ $ 6,071 ​

SEGMENT REPORTING

SEGMENT REPORTING	12 Months Ended
	Dec. 31, 2024
SEGMENT REPORTING
SEGMENT REPORTING	NOTE 11 SEGMENT REPORTING The Company has one reportable segment focused on the discovery, development, and manufacture of drug products for the treatment of various muscular disorders. Our determination that we operate as a single segment is consistent with the financial information regularly reviewed by the chief operating decision maker (CODM) for purposes of evaluating performance, allocating resources, and planning and forecasting for future periods. Our business activities are managed on a consolidated basis based on net loss that is also reported on the Statement of Operations and Comprehensive Loss as Net Loss. The Company’s CODM is the President and Chief Executive Officer (CEO). The measure of segment assets is reported on the balance sheet as total assets. ​ Substantially all of the Company’s assets are used to support the research, manufacture, and development of drug products for the treatment of muscular disorders; clinical and research data are key drivers in deciding how to allocate resources. The CEO uses net loss and significant segment expenses to monitor budget versus actual results and make decisions on whether to invest in internal or external resources to support the Company’s research and development programs, as well as determine if additional funding is needed for the Company’s research efforts. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2024 2023 Operating expenses: ​ ​ ​ ​ ​ Contracted research expense ​ $ 80,214 ​ $ 57,497 Personnel expense ​ ​ 35,797 ​ ​ 24,604 Stock-based compensation expense ​ ​ 24,712 ​ ​ 17,556 Other segment expense (a) ​ ​ 16,040 ​ ​ 13,149 Depreciation ​ ​ 2,069 ​ ​ 1,551 Segment net loss ​ ​ (158,832) ​ ​ (114,357) ​ ​ ​ ​ ​ ​ ​ Reconciliation of net loss ​ ​ ​ ​ ​ ​ Interest income ​ ​ 25,019 ​ ​ 14,194 Net Loss ​ $ (133,813) ​ $ (100,163) a Other segment expense included in Segment net loss includes contracted administrative expenses, intellectual property fees, software costs, occupancy & equipment costs, and other overhead expenses.

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (133,813)	$ (100,163)

Insider Trading Arrangements

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

Cybersecurity Risk Management a

Cybersecurity Risk Management and Strategy Disclosure	12 Months Ended
	Dec. 31, 2024
Cybersecurity Risk Management Strategy And Governance [Abstract]
Cybersecurity Risk Management Processes For Assessing Identifying And Managing Threats [Text block]	Risk Management and Strategy ​ We have established policies and processes for assessing, identifying, and managing material risk from cybersecurity threats, and have integrated these processes into our overall risk management processes. We routinely assess material risks from cybersecurity threats, including any potential unauthorized occurrence on or conducted through our information systems that may result in adverse effects on the confidentiality, integrity, or availability of our information systems or any information residing therein. We conduct periodic risk assessments to identify cybersecurity threats, as well as assessments in the event of a material change in our business practices that may affect information systems that are vulnerable to such cybersecurity threats. These risk assessments include identification of reasonably foreseeable internal and external risks, the likelihood and potential damage that could result from such risks, and the sufficiency of existing policies, procedures, systems, physical controls, and safeguards in place to manage such risks. Following these risk assessments, we evaluate whether and how to re-design, implement, and maintain reasonable safeguards to minimize identified risks and reasonably address any identified gaps in existing safeguards. We also regularly monitor the effectiveness of our safeguards. We devote significant resources and designate high-level personnel to manage the risk assessment and mitigation processes. As part of our overall risk management system, we monitor and test our safeguards and train our employees on these safeguards. Personnel at all levels and departments are made aware of our cybersecurity policies through trainings. We engage third parties in connection with our risk assessment processes. These third parties assist us to design and implement our cybersecurity policies and procedures, as well as to monitor and test our safeguards. In addition, we require each key third-party service provider to certify that it has the ability to implement and maintain appropriate security measures, consistent with all applicable laws, to implement and maintain reasonable security measures in connection with their work with us, and to promptly report any suspected breach of its security measures that may affect our company. We, like any other technology company, have experienced cybersecurity incidents in the past. However, we have not previously been materially impacted by any previous cybersecurity incidents. For additional information regarding whether any risks from cybersecurity threats are reasonably likely to materially affect our company, including our business strategy, results of operations, or financial condition, please refer to Item 1A, “Risk Factors,” in this annual report on Form 10-K.
Cybersecurity Risk Management Processes Integrated [Flag]	true
Cybersecurity Risk Management Processes Integrated [Text block]	We have established policies and processes for assessing, identifying, and managing material risk from cybersecurity threats, and have integrated these processes into our overall risk management processes. We routinely assess material risks from cybersecurity threats, including any potential unauthorized occurrence on or conducted through our information systems that may result in adverse effects on the confidentiality, integrity, or availability of our information systems or any information residing therein. We conduct periodic risk assessments to identify cybersecurity threats, as well as assessments in the event of a material change in our business practices that may affect information systems that are vulnerable to such cybersecurity threats. These risk assessments include identification of reasonably foreseeable internal and external risks, the likelihood and potential damage that could result from such risks, and the sufficiency of existing policies, procedures, systems, physical controls, and safeguards in place to manage such risks.
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 ​ One of the key functions of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats. Our board of directors is responsible for monitoring and assessing strategic risk exposure, and our executive officers are responsible for the day-to-day management of the material risks we face. Our board of directors administers its cybersecurity risk oversight function through the audit committee. Our General Counsel and our management committee on cybersecurity, which includes IT, Finance, Communications and Human Resource management, are primarily responsible to assess and manage our material risks from cybersecurity threats. Our Senior Director of IT , who is part of our management committee on cybersecurity, has been managing our cybersecurity for the last 7 years and has been certified in cybersecurity through the International Information System Security Certification Consortium. Our General Counsel and our management committee on cybersecurity oversee our cybersecurity policies and processes, including those described in “Risk Management and Strategy” above. The processes by which our General Counsel and our management committee on cybersecurity are informed about and monitor the prevention, detection, mitigation, and remediation of cybersecurity incidents include the participation of our Senior Director of IT in the management committee on cybersecurity, as well as the following: ● Prevention – management routinely takes steps to monitor compliance of our employees and third parties with the Company’s cybersecurity policies and processes. ● Detection - reports of potential cybersecurity incidents can come from various sources, including users, system operators, employees, or third parties that have noticed unusual or suspicious behavior in a system, network, or other operations processes. Employees, third parties, or authorities may report a potential incident via a telephone call, email, or other method to the organization. IT management will review the potential incident and determine whether it constitutes a potential incident and requires mitigation and notification of the management committee on cybersecurity. ● Mitigation - once a cybersecurity incident has been detected, IT management performs a vulnerability analysis and, as applicable and deemed necessary, improves system defense, removes the cause of the threat, and addresses any other vulnerabilities such as viruses, malicious codes or files, trojans, backdoors, and any authorized activity, detected. ● Remediation - remediation involves providing the technical support that is necessary to update software, repair hardware, and otherwise move our information systems toward recovery, as applicable. ​ Our General Counsel and Chief Financial Officer provide quarterly briefings to the audit committee regarding our company’s cybersecurity risks and activities, including any recent cybersecurity incidents and related responses, cybersecurity systems testing, activities of third parties, and the like. Our audit committee provides regular updates to the board of directors on such reports.
Cybersecurity Risk Board Committee Or Subcommittee Responsible For Oversight [Text block]	board of directors
Cybersecurity Risk Process For Informing Board Committee Or Subcommittee Responsible For Oversight [Text block]	One of the key functions of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats. Our board of directors is responsible for monitoring and assessing strategic risk exposure, and our executive officers are responsible for the day-to-day management of the material risks we face. Our board of directors administers its cybersecurity risk oversight function through the audit committee.
Cybersecurity Risk Role Of Management [Text block]	Our General Counsel and our management committee on cybersecurity, which includes IT, Finance, Communications and Human Resource management, are primarily responsible to assess and manage our material risks from cybersecurity threats. Our Senior Director of IT , who is part of our management committee on cybersecurity, has been managing our cybersecurity for the last 7 years and has been certified in cybersecurity through the International Information System Security Certification Consortium. Our General Counsel and our management committee on cybersecurity oversee our cybersecurity policies and processes, including those described in “Risk Management and Strategy” above. The processes by which our General Counsel and our management committee on cybersecurity are informed about and monitor the prevention, detection, mitigation, and remediation of cybersecurity incidents include the participation of our Senior Director of IT in the management committee on cybersecurity, as well as the following: ● Prevention – management routinely takes steps to monitor compliance of our employees and third parties with the Company’s cybersecurity policies and processes. ● Detection - reports of potential cybersecurity incidents can come from various sources, including users, system operators, employees, or third parties that have noticed unusual or suspicious behavior in a system, network, or other operations processes. Employees, third parties, or authorities may report a potential incident via a telephone call, email, or other method to the organization. IT management will review the potential incident and determine whether it constitutes a potential incident and requires mitigation and notification of the management committee on cybersecurity. ● Mitigation - once a cybersecurity incident has been detected, IT management performs a vulnerability analysis and, as applicable and deemed necessary, improves system defense, removes the cause of the threat, and addresses any other vulnerabilities such as viruses, malicious codes or files, trojans, backdoors, and any authorized activity, detected. ● Remediation - remediation involves providing the technical support that is necessary to update software, repair hardware, and otherwise move our information systems toward recovery, as applicable.
Cybersecurity Risk Management Positions Or Committees Responsible [Flag]	true
Cybersecurity Risk Management Positions Or Committees Responsible [Text block]	Senior Director of IT
Cybersecurity Risk Management Expertise Of Management Responsible [Text block]	Our Senior Director of IT , who is part of our management committee on cybersecurity, has been managing our cybersecurity for the last 7 years and has been certified in cybersecurity through the International Information System Security Certification Consortium.
Cybersecurity Risk Process For Informing Management Or Committees Responsible [Text block]	Our General Counsel and Chief Financial Officer provide quarterly briefings to the audit committee regarding our company’s cybersecurity risks and activities, including any recent cybersecurity incidents and related responses, cybersecurity systems testing, activities of third parties, and the like. Our audit committee provides regular updates to the board of directors on such reports.
Cybersecurity Risk Management Positions Or Committees Responsible Report To Board [Flag]	true

SUMMARY OF SIGNIFICANT ACCOUN_2

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)	12 Months Ended
	Dec. 31, 2024
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
Basis of Presentation	Basis of Presentation The accompanying financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (U.S. GAAP).
Segment Information	Segment Information The Company is organized as a single operating and reportable segment, focused on the discovery, development, and manufacture of drug products for the treatment of severe muscle diseases; segment information is presented in Note 11. All equipment and other fixed assets are physically located in the United States.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Actual results could differ from those estimates.
Cash Equivalents	Cash Equivalents The Company considers all liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents as of December 31, 2024 and 2023 primarily consist of money market funds and cash.
Concentrations of Credit Risk	Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentration of credit risk consist primarily of cash, cash equivalents and marketable securities. Periodically, the Company may maintain deposits in financial institutions in excess of government insured limits. The Company believes that it is not exposed to significant credit risk as its deposits are held at financial institutions that management believes to be of high credit quality. The Company has not experienced any losses on deposits since inception. The Company regularly invests excess cash with major financial institutions in money market funds, corporate debt securities, and commercial paper, all of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from our holdings of these financial instruments is mitigated based on the fact that many of these securities are of high credit rating.
Deferred Offering Costs	Deferred Offering Costs The Company capitalizes certain legal, professional, accounting and other third-party fees that are directly associated with in-process equity issuances as deferred offering costs until such equity issuances are consummated. After consummation of the equity issuance, these costs are recorded as a reduction in the capitalized amount associated with the equity issuance. Should the equity issuance be abandoned, the deferred offering costs are expensed immediately as a charge to operating expenses in the statement of operations. Deferred offering costs as of December 31, 2024 and December 31, 2023 were $0.3 million. Such costs are classified in other non-current assets in the accompanying balance sheets.
Property and Equipment	Property and Equipment Property and equipment is stated at cost less accumulated depreciation and amortization. Depreciation of property and equipment is computed using the straight-line method over the estimated useful life of the related asset, which is generally three
Leases	Leases The Company accounts for its leases under Accounting Standards Codification (ASC) Topic 842, Leases Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. For all asset classes of its leases, the Company has elected to account for the lease and non-lease components together for existing classes of underlying asset. Costs determined to be variable and not based on an index or rate are not included in the measurement of the lease liability.
Impairment of Long-Lived Assets	Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or circumstances indicate that the carrying value of such assets may not be fully recoverable. Impairment is evaluated based on the sum of undiscounted estimated future cash flows expected to result from use of the related asset compared to its carrying value. If impairment is recognized, the carrying value of the impaired asset is reduced to its fair value. There were no impairment charges or long-lived assets disposed of during the years ended December 31, 2024 and 2023.
Income Taxes	Income Taxes Deferred income taxes are provided on temporary differences between financial statement and income tax reporting. Temporary differences are differences between the amounts of assets and liabilities reported for financial statement purposes and their tax bases. Deferred tax assets are recognized for temporary differences that will be deductible in future years’ tax returns and for operating loss and tax credit carryforwards. Deferred tax assets are reduced by a valuation allowance if such deferred tax assets are deemed more likely than not that some or all of the deferred tax assets will not be realized. Historically, the Company has not recognized these potential benefits in its financial statements and has fully reserved for such net deferred tax assets, as it believes it is more likely than not that the full benefit of these net deferred tax assets will not be realized. Deferred tax liabilities are recognized for temporary differences that will be taxable in future years. The Company evaluated its tax positions and determined it has no uncertain tax positions as of December 31, 2024.
Fair Value of Financial Instruments	Fair Value of Financial Instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. The Financial Accounting Standards Board (FASB) ASC Topic 820, Fair Value Measurements and Disclosures Level 1—quoted prices in active markets for identical assets and liabilities. Level 2—other significant observable inputs (including quoted prices for similar assets and liabilities, interest rates, credit risk, etc.). Level 3—significant unobservable inputs (including the Company’s own assumptions in determining the fair value of assets and liabilities).
Marketable Securities, Available for Sale	Marketable Securities, Available For Sale All marketable securities have been classified as “available-for-sale” and are carried at fair value, based upon quoted market prices. The Company considers its available-for-sale portfolio as available for use in current operations. Accordingly, the Company classifies its investments as short-term marketable securities, even though the stated maturity date may be one year or more beyond the current balance sheet date. Unrealized gains and losses, net of any related tax effects, are excluded from earnings and are included in other comprehensive income (loss) and reported as a separate component of stockholders’ equity until realized. Interest income, realized gains and losses, and declines in value judged to be other than temporary, if any, on available-for-sale securities are included in other income. 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. In accordance with the Company’s investment policy, management invests in money market funds, corporate debt securities, commercial paper, asset-backed securities and government securities. The Company has not experienced any realized losses on its deposits of cash, cash equivalents, and marketable securities since inception. The following tables summarize the Company’s financial assets measured at fair value on a recurring basis by level within the fair value hierarchy (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2024 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ Hierarchy Cost Basis Gains Losses Value Cash equivalents: ​ ​ ​ ​ Money market funds Level 1 ​ $ 41,474 ​ $ — ​ $ — ​ $ 41,474 Marketable securities, available for sale: ​ ​ ​ ​ ​ ​ Asset-backed securities Level 2 ​ 50,262 ​ $ 69 ​ $ — ​ 50,331 Corporate debt securities Level 2 ​ 193,769 ​ $ 179 ​ $ (81) ​ 193,867 Commercial paper Level 2 ​ 10,432 ​ $ 3 ​ $ — ​ 10,435 U.S. government treasury and agency securities ​ Level 2 ​ 173,621 ​ $ 279 ​ $ (29) ​ 173,871 Total ​ $ 469,558 ​ $ 530 ​ $ (110) ​ $ 469,978 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2023 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ ​ Hierarchy Cost Basis Gains Losses Value Cash equivalents: ​ ​ ​ ​ Money market funds Level 1 ​ $ 85,897 ​ $ — ​ $ — ​ $ 85,897 Marketable securities, available for sale: ​ ​ ​ ​ Asset-backed securities Level 2 ​ 10,228 ​ 12 ​ (2) ​ 10,238 Corporate debt securities Level 2 ​ 82,514 ​ 66 ​ (113) ​ 82,467 Commercial paper ​ Level 2 ​ ​ 19,457 ​ ​ 13 ​ ​ (8) ​ ​ 19,462 U.S. government treasury and agency securities ​ Level 2 ​ ​ 116,579 ​ ​ 151 ​ ​ (26) ​ ​ 116,704 Supranational and sovereign government securities Level 2 ​ 3,419 ​ 6 ​ — ​ 3,425 Total ​ $ 318,094 ​ $ 248 ​ $ (149) ​ $ 318,193 ​ The Company’s money market funds are classified as Level 1 because they are valued using quoted market prices. Investments in asset-backed securities, corporate debt securities, commercial paper and U.S. government treasury and agency securities, and supranational and sovereign government securities have been classified as Level 2 as they are valued using quoted prices in less active markets or other directly or indirectly observable inputs. Fair values of asset-backed securities, corporate debt securities, commercial paper, U.S. government treasury and agency securities, and supranational and sovereign government securities were derived based on input of market prices from multiple sources at each reporting period. With regard to commercial paper, all of the securities had high credit ratings and one year or less to maturity; therefore, fair value was derived from accretion of purchase price to face value over the term of maturity or quoted market prices for similar instruments if available. There were no transfers of financial assets between Level 1, Level 2, or Level 3, during the periods presented. As of December 31, 2024, remaining contractual maturities of $394.4 million of marketable securities were less than one year 1 The Company periodically reviews its portfolio of debt securities to determine if any investment is impaired due to credit loss or other potential valuation concerns. For debt securities where the fair value of the investment is less than the amortized cost basis, the Company has assessed at the individual security level for various quantitative factors including, but not limited to, the nature of the investments, changes in credit ratings, interest rate fluctuations, industry analyst reports, and the severity of impairment. Unrealized losses on marketable securities at December 31, 2024 were primarily due to changes in interest rates, including market credit spreads, and not due to increased credit risks associated with specific securities.
Comprehensive Income (Loss)	Comprehensive Income (Loss) Comprehensive income (loss) is defined as the change in equity during a period from transactions and other events and/or circumstances from non-owner sources. The Company’s only element of other comprehensive income (loss) was net unrealized gain (loss) on marketable securities.
Stock-Based Compensation	Stock-Based Compensation In accordance with ASC Topic 718, Compensation—Stock Compensation
Research and Development Expenses and Accrued Research and Development Expenses	Research and Development Expenses and Accrued Research and Development Expenses Expenditures made for research and development are charged to expense as incurred. External costs consist primarily of payments to contract research organizations (CROs), contract development and manufacturing organizations (CDMOs), sample acquisition costs and laboratory supplies purchased in connection with the Company’s discovery and preclinical activities, and process development and clinical development activities. Internal costs consist primarily of employee-related costs, facilities, depreciation and costs related to compliance with regulatory requirements. Non-refundable advance payments for goods and services that will be used in future research and development activities are capitalized and recorded as an expense in the period that the Company receives the goods or when services are performed. The Company records expenses related to external research and development services based on its estimates of the services received and efforts expended pursuant to quotes and contracts with multiple CROs and CDMOs that supply, conduct and manage preclinical studies and clinical trials on its behalf. The financial terms of these contracts vary from contract to contract and may result in payment flows that do not match the periods over which materials or services are provided under such contracts. 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. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company adjusts the accrual or the amount of prepaid expenses accordingly.
Recently Adopted Accounting Pronouncements	Recently Adopted Accounting Pronouncements In June 2016, the FASB issued Accounting Standards Update (ASU) 2016-13, Financial Instruments - Credit Losses: Measurement of Credit Losses on Financial Statements ​ In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures
Accounting Standards Not Yet Adopted	Accounting Standards Not Yet Adopted In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures In November 2024, the FASB issued ASU 2024-03, Income Statement–Reporting Comprehensive Income–Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses

SUMMARY OF SIGNIFICANT ACCOUN_3

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables)	12 Months Ended
	Dec. 31, 2024
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
Schedule of financial assets measured at fair value on a recurring basis by level within the fair value hierarchy	The following tables summarize the Company’s financial assets measured at fair value on a recurring basis by level within the fair value hierarchy (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2024 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ Hierarchy Cost Basis Gains Losses Value Cash equivalents: ​ ​ ​ ​ Money market funds Level 1 ​ $ 41,474 ​ $ — ​ $ — ​ $ 41,474 Marketable securities, available for sale: ​ ​ ​ ​ ​ ​ Asset-backed securities Level 2 ​ 50,262 ​ $ 69 ​ $ — ​ 50,331 Corporate debt securities Level 2 ​ 193,769 ​ $ 179 ​ $ (81) ​ 193,867 Commercial paper Level 2 ​ 10,432 ​ $ 3 ​ $ — ​ 10,435 U.S. government treasury and agency securities ​ Level 2 ​ 173,621 ​ $ 279 ​ $ (29) ​ 173,871 Total ​ $ 469,558 ​ $ 530 ​ $ (110) ​ $ 469,978 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, 2023 ​ ​ Fair Value ​ Amortized ​ Unrealized ​ Unrealized ​ Fair Market ​ ​ Hierarchy Cost Basis Gains Losses Value Cash equivalents: ​ ​ ​ ​ Money market funds Level 1 ​ $ 85,897 ​ $ — ​ $ — ​ $ 85,897 Marketable securities, available for sale: ​ ​ ​ ​ Asset-backed securities Level 2 ​ 10,228 ​ 12 ​ (2) ​ 10,238 Corporate debt securities Level 2 ​ 82,514 ​ 66 ​ (113) ​ 82,467 Commercial paper ​ Level 2 ​ ​ 19,457 ​ ​ 13 ​ ​ (8) ​ ​ 19,462 U.S. government treasury and agency securities ​ Level 2 ​ ​ 116,579 ​ ​ 151 ​ ​ (26) ​ ​ 116,704 Supranational and sovereign government securities Level 2 ​ 3,419 ​ 6 ​ — ​ 3,425 Total ​ $ 318,094 ​ $ 248 ​ $ (149) ​ $ 318,193

STOCK-BASED COMPENSATION AWAR_2

STOCK-BASED COMPENSATION AWARDS (Tables)	12 Months Ended
	Dec. 31, 2024
STOCK-BASED COMPENSATION AWARDS
Schedule of option activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Average ​ ​ ​ ​ Weighted ​ ​ ​ ​ Remaining ​ ​ ​ ​ Average ​ Total Intrinsic ​ Contractual Life ​ Options Exercise Price Value (000's) (Years) Outstanding as of December 31, 2023 14,491,613 $ 7.43 $ 65,801 ​ 8.0 Granted 2,817,497 ​ $ 17.67 ​ ​ ​ ​ ​ Exercised (1,621,074) ​ $ 6.00 ​ ​ ​ ​ ​ Cancelled ​ (271,275) ​ $ 10.41 ​ ​ ​ ​ ​ Outstanding as of December 31, 2024 ​ 15,416,761 ​ $ 9.40 ​ $ 267,397 ​ 7.5 Options exercisable as of December 31, 2024 ​ 8,643,539 ​ $ 7.13 ​ $ 169,659 ​ 6.6
Schedule of fair value assumptions	​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 Expected term (Years) ​ 5.10 - 6.85 ​ 5.44 - 6.85 Expected volatility ​ 78.67% - 83.21% ​ 80.68% - 85.68% Risk-free interest rate ​ 3.59% - 4.66% ​ 3.47% - 4.80% Expected dividend rate ​ - ​ - Fair value common stock ​ $16.33 - $33.57 ​ $5.73 - $10.29
Schedule of unvested restricted stock units	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted Average ​ ​ ​ ​ Grant Date ​ RSUs Fair Value Issued and unvested as of December 31, 2023 177,871 $ 7.98 Granted 557,027 ​ $ 17.05 Vested (84,855) ​ $ 7.98 Cancelled ​ (10,527) ​ $ 10.03 Issued and unvested as of December 31, 2024 ​ 639,516 ​ $ 15.85
Schedule of stock-based compensation expense	Total stock-based compensation expense related to all equity plans, including Founder Stock Options was allocated as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 Research and development $ 13,956 $ 10,029 General and administrative ​ 10,755 ​ 7,531 Total stock-based compensation expense ​ $ 24,711 ​ $ 17,560 ​

COMMITMENTS AND CONTINGENCIES (

COMMITMENTS AND CONTINGENCIES (Tables)	12 Months Ended
	Dec. 31, 2024
Boulder Lease
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Summary of maturities of leases	Future minimum lease payments under the Lease as of December 31, 2024 are as follows (in thousands): ​ ​ ​ ​ ​ Year Ending December 31, ​ ​ ​ 2025 $ 1,031 2026 ​ ​ 1,048 2027 ​ 1,066 2028 ​ ​ 1,084 2029 ​ ​ 1,103 Thereafter ​ ​ 278 Total undiscounted future minimum lease payments ​ ​ 5,610 Less: discount ​ ​ (873) Total lease liability ​ $ 4,737

INCOME TAXES (Tables)

INCOME TAXES (Tables)	12 Months Ended
	Dec. 31, 2024
INCOME TAXES
Schedule of the effective income tax rate	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ 2024 2023 Federal tax at statutory rate ​ 21.0 % ​ 21.0 % Research and development credits ​ 5.6 % ​ 5.8 % State taxes, net of federal deduction ​ 5.5 % ​ 4.9 % Other ​ 3.4 % ​ 0.1 % Disallowed compensation ​ (4.0) % ​ (1.8) % Change in valuation allowance ​ (31.5) % ​ (30.0) % Effective income tax rate ​ — ​ — ​
Schedule of deferred tax assets and liabilities	Deferred tax assets and liabilities are as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ 2024 2023 Deferred tax assets (liabilities): ​ ​ ​ ​ ​ ​ Capitalized research expenses ​ $ 49,054 ​ $ 25,316 Federal net operating loss carryforward ​ ​ 30,714 ​ ​ 22,971 Federal and state R&D credits ​ ​ 16,854 ​ ​ 8,913 State net operating loss carryforward ​ ​ 6,956 ​ ​ 5,197 Stock-based compensation ​ ​ 4,929 ​ ​ 4,120 Accrued expenses ​ ​ 1,661 ​ ​ 1,499 Lease liability ​ ​ 1,236 ​ ​ 1,411 Orphan Drug Credit ​ ​ 606 ​ ​ 691 Property and equipment ​ ​ 107 ​ ​ 30 Other ​ ​ (15) ​ ​ 53 ROU asset ​ ​ (410) ​ ​ (586) Total net deferred tax asset ​ ​ 111,692 ​ ​ 69,615 Valuation allowance ​ ​ (111,692) ​ ​ (69,615) Net deferred tax asset (liability) ​ $ — ​ $ —

NET LOSS PER SHARE (Tables)

NET LOSS PER SHARE (Tables)	12 Months Ended
	Dec. 31, 2024
NET LOSS PER SHARE
Schedule of computation of the basic and diluted net loss per share	The following table sets forth the computation of the basic and diluted net loss per share (in thousands, except share and per share data): ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2024 2023 Numerator ​ ​ ​ ​ ​ ​ Net loss ​ $ (133,813) $ (100,163) Denominator ​ ​ ​ ​ Weighted-average shares outstanding used in computing net loss per share, basic and diluted ​ ​ 92,414,626 ​ ​ 63,723,600 Net loss per share, basic and diluted ​ $ (1.45) ​ $ (1.57)
Schedule of potentially dilutive securities	The following weighted average outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2024 2023 Options to purchase common stock ​ ​ 16,340,740 ​ ​ 13,764,188 Unvested restricted stock units ​ ​ 328,984 ​ ​ 207,628 Total ​ ​ 16,669,724 ​ ​ 13,971,816 ​

PROPERTY AND EQUIPMENT (Tables)

PROPERTY AND EQUIPMENT (Tables)	12 Months Ended
	Dec. 31, 2024
PROPERTY AND EQUIPMENT
Schedule of property plant and equipment	Property and equipment consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ As of December 31, ​ 2024 2023 Leasehold improvements ​ $ 9,646 ​ $ 8,728 Laboratory equipment ​ ​ 3,735 ​ ​ 3,473 Computers and software ​ ​ 296 ​ ​ 281 Furniture and fixtures ​ ​ 511 ​ ​ 497 Construction in process ​ ​ — ​ ​ 80 Property and equipment, at cost ​ ​ 14,188 ​ ​ 13,059 Less: accumulated depreciation ​ ​ (4,685) ​ ​ (2,616) Property and equipment, net ​ $ 9,503 ​ $ 10,443

ACCRUED OTHER EXPENSES (Tables)

ACCRUED OTHER EXPENSES (Tables)	12 Months Ended
	Dec. 31, 2024
ACCRUED OTHER EXPENSES
Schedule of accrued other expenses	Accrued other expenses consisted of the following amounts (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ As of December 31, ​ As of December 31, ​ 2024 2023 Accrued research and development costs ​ $ 6,488 ​ $ 5,672 Accrued other ​ ​ 741 ​ ​ 399 Total accrued other expenses ​ $ 7,229 ​ $ 6,071 ​

SEGMENT REPORTING (Tables)

SEGMENT REPORTING (Tables)	12 Months Ended
	Dec. 31, 2024
SEGMENT REPORTING
Schedule of revenue and expenses for the operating segment to consolidated amounts	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year Ended December 31, ​ ​ 2024 2023 Operating expenses: ​ ​ ​ ​ ​ Contracted research expense ​ $ 80,214 ​ $ 57,497 Personnel expense ​ ​ 35,797 ​ ​ 24,604 Stock-based compensation expense ​ ​ 24,712 ​ ​ 17,556 Other segment expense (a) ​ ​ 16,040 ​ ​ 13,149 Depreciation ​ ​ 2,069 ​ ​ 1,551 Segment net loss ​ ​ (158,832) ​ ​ (114,357) ​ ​ ​ ​ ​ ​ ​ Reconciliation of net loss ​ ​ ​ ​ ​ ​ Interest income ​ ​ 25,019 ​ ​ 14,194 Net Loss ​ $ (133,813) ​ $ (100,163) a Other segment expense included in Segment net loss includes contracted administrative expenses, intellectual property fees, software costs, occupancy & equipment costs, and other overhead expenses.

DESCRIPTION OF BUSINESS (Detail

DESCRIPTION OF BUSINESS (Details) - USD ($)					12 Months Ended
	May 10, 2024	Jan. 23, 2024	Jan. 19, 2024	Jun. 16, 2023	Dec. 31, 2024	Dec. 31, 2023
Subsidiary, Sale of Stock [Line Items]
Accumulated deficit					$ 378,576,000	$ 244,763,000
Cash, cash equivalents and marketable securities					470,200,000
Proceeds from issuance of common stock					$ 239,147,000	$ 52,618,000
Maximum
Subsidiary, Sale of Stock [Line Items]
Share price					$ 33.57	$ 10.29
Common Stock
Subsidiary, Sale of Stock [Line Items]
Shares issued					22,450,206	6,928,044
Underwriters' option
Subsidiary, Sale of Stock [Line Items]
Shares issued		21,818,182
Share price		$ 11
Gross proceeds		$ 240,000,000
Proceeds from issuance of common stock		231,900,000
Underwriting discounts and commissions		7,500,000
Offering expenses		$ 600,000
At-the-market program
Subsidiary, Sale of Stock [Line Items]
Shares issued			7,560,068
Share price			$ 7.93
Gross proceeds			$ 59,900,000
Proceeds from issuance of common stock			59,400,000
Underwriting discounts and commissions			200,000
Offering expenses			$ 300,000
At-the-market program | BofA securities, Inc | Maximum
Subsidiary, Sale of Stock [Line Items]
Proceeds from issuance of common stock				$ 125,000,000
At-the-market program | Common Stock | Maximum
Subsidiary, Sale of Stock [Line Items]
Maximum potential aggregate sales proceeds, sales agreement	$ 175,000,000

SUMMARY OF SIGNIFICANT ACCOUN_4

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Impairment of Long-Lived Assets
Impairment of long-lived assets	$ 0	$ 0
Income Taxes.
Uncertain tax positions	$ 0
Minimum
Property and Equipment
Estimated useful life	3 years
Maximum
Property and Equipment
Estimated useful life	7 years
Other non-current assets
Deferred Offering Costs
Deferred offering costs	$ 300	$ 300

SUMMARY OF SIGNIFICANT ACCOUN_5

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Marketable Securities, Available For Sale
Cash and cash equivalents	$ 41,666	$ 86,097
Fair Value, Recurring
Marketable Securities, Available For Sale
Amortized Cost Basis, Marketable securities, available for sale	469,558	318,094
Unrealized Gains, Marketable securities, available for sale	530	248
Unrealized Losses, Marketable securities, available for sale	(110)	(149)
Fair Market Value, Marketable securities, available for sale	469,978	318,193
Fair Value, Recurring | Level 1 | Money market funds
Marketable Securities, Available For Sale
Cash and cash equivalents	41,474	85,897
Cash equivalents, Fair Market Value	41,474	85,897
Fair Value, Recurring | Level 2 | Asset-backed securities
Marketable Securities, Available For Sale
Amortized Cost Basis, Marketable securities, available for sale	50,262	10,228
Unrealized Gains, Marketable securities, available for sale	69	12
Unrealized Losses, Marketable securities, available for sale		(2)
Fair Market Value, Marketable securities, available for sale	50,331	10,238
Fair Value, Recurring | Level 2 | Corporate debt securities
Marketable Securities, Available For Sale
Amortized Cost Basis, Marketable securities, available for sale	193,769	82,514
Unrealized Gains, Marketable securities, available for sale	179	66
Unrealized Losses, Marketable securities, available for sale	(81)	(113)
Fair Market Value, Marketable securities, available for sale	193,867	82,467
Fair Value, Recurring | Level 2 | Commercial paper
Marketable Securities, Available For Sale
Amortized Cost Basis, Marketable securities, available for sale	10,432	19,457
Unrealized Gains, Marketable securities, available for sale	3	13
Unrealized Losses, Marketable securities, available for sale		(8)
Fair Market Value, Marketable securities, available for sale	10,435	19,462
Fair Value, Recurring | Level 2 | U.S. government treasury and agency securities
Marketable Securities, Available For Sale
Amortized Cost Basis, Marketable securities, available for sale	173,621	116,579
Unrealized Gains, Marketable securities, available for sale	279	151
Unrealized Losses, Marketable securities, available for sale	(29)	(26)
Fair Market Value, Marketable securities, available for sale	$ 173,871	116,704
Fair Value, Recurring | Level 2 | Supranational and sovereign government securities
Marketable Securities, Available For Sale
Amortized Cost Basis, Marketable securities, available for sale		3,419
Unrealized Gains, Marketable securities, available for sale		6
Fair Market Value, Marketable securities, available for sale		$ 3,425

SUMMARY OF SIGNIFICANT ACCOUN_6

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Financial Assets Transfers and Maturities (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Remaining contractual maturities
Fair value assets transfer from level 1 to 2	$ 0	$ 0
Fair value assets transfer from level 2 to 1	0	0
Fair value assets transfer in/out of level 3	0	$ 0
Less than one year	394,400
One year through two years	$ 33,700
Minimum
Remaining contractual maturities
Maturity Period	1 year
Maximum
Remaining contractual maturities
Maturity Period	2 years

PREFERRED STOCK AND COMMON ST_2

PREFERRED STOCK AND COMMON STOCK - Narrative (Details)	Dec. 31, 2024 Vote $ / shares shares	Dec. 31, 2023 $ / shares shares
PREFERRED STOCK AND COMMON STOCK
Total shares authorized	1,200,000,000
Common stock authorized (in shares)	1,000,000,000	1,000,000,000
Preferred stock, shares authorized (in shares)	200,000,000	200,000,000
Par value of common stock (in dollars per share) | $ / shares	$ 0.0001	$ 0.0001
Par value of preferred stock (in dollars per share) | $ / shares	$ 0.0001	$ 0.0001
Voting rights upon conversion into common stock | Vote	1

STOCK-BASED COMPENSATION AWAR_3

STOCK-BASED COMPENSATION AWARDS - Equity Incentive Plans (Details) - USD ($) $ / shares in Units, $ in Millions	1 Months Ended	12 Months Ended
	Mar. 31, 2021	Dec. 31, 2024	Dec. 31, 2023	Aug. 10, 2024
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Option exercised term		2 years 8 months 12 days
RSU
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Restricted stock unit granted (in shares)		557,027
Option exercised term		3 years 6 months
Unrecognized stock-based compensation cost		$ 8.7
RSU | Minimum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting of stock options (in years)		3 years
RSU | Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting of stock options (in years)		4 years
2024 Inducement Equity Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares reserved for the issuance of stock options				2,000,000
Shares available for future issuance		2,000,000
2021 Equity Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting of stock options (in years)	4 years
Expiration period (in years)	10 years
Shares available for future issuance		1,453,866
Percentage of outstanding shares		5%
Options granted (in shares)		2,817,497
Unrecognized stock-based compensation cost		$ 56.1
Aggregate intrinsic value of options exercised		$ 30.3	$ 1
Weighted-average grant date fair value (in dollars per share)		$ 12.73	$ 5.25
2021 Equity Incentive Plan | Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance		5,040,000
2021 Equity Incentive Plan | Employees, Directors and Consultants
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares reserved for the issuance of stock options		5,040,000

STOCK-BASED COMPENSATION AWAR_4

STOCK-BASED COMPENSATION AWARDS - Options Activity (Details) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2024 USD ($) $ / shares shares
Total Intrinsic Value
Intrinsic Value of outstanding options | $	$ 65,801
Weighted average remaining contractual life of outstanding options (in years)	8 years
2021 Equity Incentive Plan
Options
Outstanding as at beginning of period (in shares) | shares	14,491,613
Granted (in shares) | shares	2,817,497
Exercised (in shares) | shares	(1,621,074)
Cancelled (in shares) | shares	(271,275)
Outstanding as at end of period (in shares) | shares	15,416,761
Options exercisable as at end of period (in shares) | shares	8,643,539
Weighted Average Exercise Price
Outstanding as at beginning of period (in dollars per shares) | $ / shares	$ 7.43
Granted (in dollars per shares) | $ / shares	17.67
Exercised (in dollars per shares) | $ / shares	6
Cancelled (in dollars per shares) | $ / shares	10.41
Outstanding as at end of period (in dollars per shares) | $ / shares	9.40
Options exercisable as at end of period (in dollars per shares) | $ / shares	$ 7.13
Total Intrinsic Value
Intrinsic Value of outstanding options | $	$ 267,397
Intrinsic value of exercisable options (in dollars) | $	$ 169,659
Weighted average remaining contractual life of outstanding options (in years)	7 years 6 months
Weighted average remaining contractual life of exercisable options (in years)	6 years 7 months 6 days

STOCK-BASED COMPENSATION AWAR_5

STOCK-BASED COMPENSATION AWARDS - Founder Stock Options (Details) - USD ($) $ / shares in Units, $ in Thousands		12 Months Ended
	Sep. 19, 2017	Dec. 31, 2024
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Intrinsic Value of outstanding options		$ 65,801
Founder Stock Options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Options granted (in shares)	1,795,880
Exercise price (in dollars per share)	$ 0.18
Vesting period (in years)	4 years
Expiration period (in years)	15 years
Options exercisable (in shares)		1,356,780
Options outstanding (in shares)		1,356,780
Exercise of stock options (shares)		175,000
Weighted average remaining contractual life of exercisable options (in years)		7 years 6 months
Intrinsic Value of outstanding options		$ 4,600
Intrinsic value of exercisable options		$ 36,000

STOCK-BASED COMPENSATION AWAR_6

STOCK-BASED COMPENSATION AWARDS - Assumptions (Details) - $ / shares	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected term (Years)	6 years 10 months 6 days	6 years 10 months 6 days
Expected volatility	83.21%	85.68%
Risk-free interest rate	4.66%	4.80%
Fair value common stock	$ 33.57	$ 10.29
Minimum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected term (Years)	5 years 1 month 6 days	5 years 5 months 8 days
Expected volatility	78.67%	80.68%
Risk-free interest rate	3.59%	3.47%
Fair value common stock	$ 16.33	$ 5.73

STOCK-BASED COMPENSATION AWAR_7

STOCK-BASED COMPENSATION AWARDS - Restricted Stock Units (Details) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2024 USD ($) item $ / shares shares	Dec. 31, 2023 USD ($) $ / shares shares
Grant Date Fair Value
Period over which cost is expected to be recognized (in years)	2 years 8 months 12 days
RSU
RSUs
Issued and unvested (in shares) | shares	177,871
Restricted stock unit granted (in shares) | shares	557,027
Vested (in shares) | shares	(84,855)
Cancelled | shares	(10,527)
Issued and unvested (in shares) | shares	639,516	177,871
Grant Date Fair Value
Issued and unvested (in dollars per share) | $ / shares	$ 7.98
Granted (in dollars per share) | $ / shares	17.05
Vested (in dollars per share) | $ / shares	7.98
Cancelled (in dollars per share) | $ / shares	10.03
Issued and unvested (in dollars per share) | $ / shares	$ 15.85	$ 7.98
Unrecognized stock-based compensation cost | $	$ 8.7
Period over which cost is expected to be recognized (in years)	3 years 6 months
Number of common stock issued upon vesting of RSU | item	1
Total fair value | $	$ 0.7	$ 0.7
RSU | Minimum
Grant Date Fair Value
Vesting period (in years)	3 years
RSU | Maximum
Grant Date Fair Value
Vesting period (in years)	4 years

STOCK-BASED COMPENSATION AWAR_8

STOCK-BASED COMPENSATION AWARDS - Employee Stock Purchase Plan (Details) - 2021 Employee Stock Purchase Plan	12 Months Ended
	Dec. 31, 2024 period $ / shares shares	Dec. 31, 2023 $ / shares shares
Employee Stock Ownership Plan (ESOP) Disclosures [Line Items]
Shares initially reserved for issuance	504,000
Percentage of outstanding shares	1%
Shares reserved for issuance of common stock	2,027,407
Number of offering periods | period	2
Maximum percentage of contributions under ESPP	15%
Maximum number of shares per employee (in shares)	6,000
Discounted purchase price in relation to market price (percent)	85%
Purchase of common stock under employee stock purchase plan (in shares)	147,086	95,259
Maximum
Employee Stock Ownership Plan (ESOP) Disclosures [Line Items]
Shares available for future issuance	1,008,000
Weighted average
Employee Stock Ownership Plan (ESOP) Disclosures [Line Items]
Offering price per share (in dollars per share) | $ / shares	$ 6.03	$ 6.26

STOCK-BASED COMPENSATION AWAR_9

STOCK-BASED COMPENSATION AWARDS - 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 stock-based compensation expense	$ 24,711	$ 17,560
Research and development
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	13,956	10,029
General and administrative
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	$ 10,755	$ 7,531

COMMITMENTS AND CONTINGENCIES_2

COMMITMENTS AND CONTINGENCIES (Details) $ in Thousands	1 Months Ended	12 Months Ended
	Jan. 31, 2022 USD ($) ft²	Dec. 31, 2024 USD ($) item	Dec. 31, 2023 USD ($)	Feb. 28, 2023 USD ($) ft²
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Increase in operating lease liabilities		$ 2,000
Boulder Lease
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Area of property under lease | ft²	18,614			9,624
Aggregate lease amount payable over the lease term	$ 3,300	$ 5,610		$ 1,500
Term of lease (in years)	8 years 2 months 12 days
Initial term of no default condition for replacement of letter of credit	36 months
Number of tenant improvement allowances | item		2
Tenant improvement allowance				$ 500
Increase in aggregate base rent payments		$ 2,500
Amount received by the lessor		$ 500
Number of terms option to extend the lease | item		2
Number of years in additional terms in option to extend the lease		5 years
Maximum percentage of net rent, payable to Lessor for operating costs		5%
Variable lease cost		$ 500	$ 300
Weighted-average remaining lease term		5 years 3 months 18 days		7 years 3 months 18 days
Weighted-average discount rate		6.40%
Rent expense		$ 500	$ 500
Boulder Lease | Letter of credit
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Amount of Standby Letter of Credit	$ 800
Boulder Lease | No Event of Default During Initial 36-Month Lease Term | Letter of credit
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Amount of Standby Letter of Credit	500
Boulder Lease | First allowance
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Tenant improvement allowance	1,000
Boulder Lease | Second allowance
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Tenant improvement allowance	$ 2,000

COMMITMENTS AND CONTINGENCIES -

COMMITMENTS AND CONTINGENCIES - Future minimum lease payments (Details) - Boulder Lease - USD ($) $ in Thousands	Dec. 31, 2024	Feb. 28, 2023	Jan. 31, 2022
Future minimum lease payment
2025	$ 1,031
2026	1,048
2027	1,066
2028	1,084
2029	1,103
Thereafter	278
Total undiscounted future minimum lease payments	5,610	$ 1,500	$ 3,300
Less: discount	(873)
Total lease liability	$ 4,737

INCOME TAXES - Effective income

INCOME TAXES - Effective income tax rate reconciliation (Details)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
INCOME TAXES
Federal tax at statutory rate	21%	21%
Research and development credits	5.60%	5.80%
State taxes, net of federal deduction	5.50%	4.90%
Other	3.40%	0.10%
Disallowed compensation	(4.00%)	(1.80%)
Change in valuation allowance	(31.50%)	(30.00%)
Effective income tax rate

INCOME TAXES - Deferred Tax Ass

INCOME TAXES - Deferred Tax Assets (Liabilities) (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Deferred tax assets (liabilities):
Capitalized research expenses	$ 49,054	$ 25,316
Federal net operating loss carryforward	30,714	22,971
Federal and state R&D credits	16,854	8,913
State net operating loss carryforward	6,956	5,197
Stock-based compensation	4,929	4,120
Accrued expenses	1,661	1,499
Lease liability	1,236	1,411
Orphan Drug Credit	606	691
Property and equipment	107	30
Other	(15)	53
ROU asset	(410)	(586)
Total net deferred tax asset	111,692	69,615
Valuation allowance	(111,692)	(69,615)
Net deferred tax asset (liability)

INCOME TAXES - Narrative (Detai

INCOME TAXES - Narrative (Details)	12 Months Ended
	Dec. 31, 2024 USD ($)
Operating Loss Carryforwards [Line Items]
Provision for income taxes	$ 0
Federal
Operating Loss Carryforwards [Line Items]
Net operating loss carryforwards	146,300,000
Amount of limited carryfoward	1,200,000
Amount of indefinite carryforward	145,100,000
Federal | Research tax credit carryforward
Operating Loss Carryforwards [Line Items]
Tax credits carryforwards	16,600,000
State
Operating Loss Carryforwards [Line Items]
Net operating loss carryforwards	155,600,000
Amount of limited carryfoward	122,900,000
Amount of indefinite carryforward	32,700,000
State | Research tax credit carryforward
Operating Loss Carryforwards [Line Items]
Amount of limited carryfoward	500,000
Amount of indefinite carryforward	400,000
Tax credits carryforwards	$ 900,000

EMPLOYEE BENEFIT PLANS (Details

EMPLOYEE BENEFIT PLANS (Details) - USD ($) $ in Millions	1 Months Ended	12 Months Ended
	Sep. 30, 2021	Dec. 31, 2024	Dec. 31, 2023
Defined Contribution Plan Disclosure [Line Items]
Matching contributions		$ 1	$ 0.6
Maximum
Defined Contribution Plan Disclosure [Line Items]
Percentage of compensation	4%

NET LOSS PER SHARE - Computatio

NET LOSS PER SHARE - Computation of Net loss per share (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Numerator
Net loss	$ (133,813)	$ (100,163)
Denominator
Weighted-average shares outstanding used in computing net loss per share, basic	92,414,626	63,723,600
Weighted-average shares outstanding used in computing net loss per share, diluted	92,414,626	63,723,600
Net loss per share, basic	$ (1.45)	$ (1.57)
Net loss per share, diluted	$ (1.45)	$ (1.57)

NET LOSS PER SHARE - Potentiall

NET LOSS PER SHARE - Potentially dilutive securities (Details) - shares	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share
Potentially dilutive securities (in shares)	16,669,724	13,971,816
Employee Stock Option [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share
Potentially dilutive securities (in shares)	16,340,740	13,764,188
RSU
Antidilutive Securities Excluded from Computation of Earnings Per Share
Potentially dilutive securities (in shares)	328,984	207,628

PROPERTY AND EQUIPMENT (Details

PROPERTY AND EQUIPMENT (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Property, Plant and Equipment [Line Items]
Property and equipment, at cost	$ 14,188	$ 13,059
Less: accumulated depreciation	(4,685)	(2,616)
Property and equipment, net	9,503	10,443
Depreciation expense	2,069	1,551
Leasehold improvements
Property, Plant and Equipment [Line Items]
Property and equipment, at cost	9,646	8,728
Laboratory equipment
Property, Plant and Equipment [Line Items]
Property and equipment, at cost	3,735	3,473
Computers and software
Property, Plant and Equipment [Line Items]
Property and equipment, at cost	296	281
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Property and equipment, at cost	$ 511	497
Construction in process
Property, Plant and Equipment [Line Items]
Property and equipment, at cost		$ 80

ACCRUED OTHER EXPENSES (Details

ACCRUED OTHER EXPENSES (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
ACCRUED OTHER EXPENSES
Accrued research and development costs	$ 6,488	$ 5,672
Accrued other	741	399
Total accrued other expenses	$ 7,229	$ 6,071

SEGMENT REPORTING (Details)

SEGMENT REPORTING (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2024 USD ($) item	Dec. 31, 2023 USD ($)
Segment Reporting Information [Line Items]
Number of reportable segments | item	1
Operating expenses:
Stock-based compensation expense	$ 24,711	$ 17,560
Loss from operations	(158,832)	(114,357)
Net loss	(133,813)	(100,163)
Single reportable segment
Operating expenses:
Contracted research expense	80,214	57,497
Personnel expense	35,797	24,604
Stock-based compensation expense	24,712	17,556
Other segment expense	16,040	13,149
Depreciation	2,069	1,551
Loss from operations	(158,832)	(114,357)
Interest income	25,019	14,194
Net loss	$ (133,813)	$ (100,163)