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CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)
$ in Thousands
 12 Months Ended
Dec. 31, 2023
Dec. 31, 2022
Dec. 31, 2021
Operating activities      
Net loss $ (184,678) $ (181,119) $ (69,612)
Adjustments to reconcile net loss to net cash used in operating activities:      
Stock-based compensation expense 32,553 19,831 20,804
Gain on sale of priority review voucher     (100,000)
Depreciation and amortization 1,758 1,672 1,158
Non-cash interest expense and amortization of debt issuance costs 13,360 5,389  
Non-cash accretion & amortization of short-term investments (9,835) (2,314)  
Non-cash rent expense 401 (267) (250)
Loss on RareStone equity investment   1,040  
Change in fair value of embedded derivative liability (190) (250)  
Acquired IPR&D assets classified as investing activities 5,667    
Changes in operating assets and liabilities:      
Accounts receivable (8,643) (5,199) (1,250)
Inventory (5,707) (2,806) (11)
Prepaid expenses and other current assets 2,876 498 (3,339)
Deferred revenue (148) (6,606)  
Other long-term assets, net 1,656 (4,840) (11,815)
Accounts payable, accrued expenses and other liabilities 14,773 1,543 18,312
Net cash used in operating activities (136,157) (173,428) (146,003)
Investing activities      
Purchases of short-term investments (354,918) (251,937) (524,972)
Maturities of short-term investments 354,967 284,247 361,247
Proceeds from sale of priority review voucher     100,000
Proceeds from out-license agreement     7,000
Payment of milestone obligation under license agreement   (4,000) (5,000)
Acquisition of IPR&D assets, including transaction costs (5,667)    
Purchases of property and equipment (47) (281) (434)
Net cash (used in) provided by investing activities (5,665) 28,029 (62,159)
Financing activities      
Repayment of deferred royalty obligation (7,398)    
Net proceeds from issuance of common stock 48,875 131,112 161,726
Proceeds from the exercise of stock options 7,468 9,752 4,134
Proceeds from issuance of common stock from ESPP 1,053 626 621
Proceeds from royalty financing agreement, net of issuance costs 24,370 72,338  
Net cash provided by financing activities 74,368 213,828 166,481
Effect of exchange rates on cash (142)    
Net (decrease) increase in cash, cash equivalents and restricted cash (67,596) 68,429 (41,681)
Cash, cash equivalents and restricted cash at beginning of period 128,005 59,576 101,257
Cash, cash equivalents and restricted cash at end of period $ 60,409 $ 128,005 $ 59,576