v3.26.1
Segment Information - Summary of Segment Information (Details) - USD ($)
 3 Months Ended 6 Months Ended
Jun. 30, 2026
Mar. 31, 2026
Jun. 30, 2025
Mar. 31, 2025
Jun. 30, 2026
Jun. 30, 2025
Segment Reporting [Line Items]            
Revenue $ 9,325,000   $ 13,817,000   $ 15,554,000 $ 172,386,000
Operating expenses (74,754,000)   (41,117,000)   (134,401,000) (88,446,000)
Interest expense (17,000)   (3,000)   (41,000) (3,000)
Interest income 3,520,000   3,792,000   6,939,000 6,681,000
Other income 308,000   28,000   327,000 57,000
Provision for income taxes 0   0   0 (1,278,000)
Net (loss) income (61,618,000) $ (50,003,000) (23,483,000) $ 112,879,000 (111,621,000) 89,397,000
Zorevunersen            
Segment Reporting [Line Items]            
Operating expenses [1] 28,243,000   11,382,000   48,097,000 29,056,000
ADOA            
Segment Reporting [Line Items]            
Operating expenses [1] 2,697,000   1,747,000   4,555,000 2,140,000
SYNGAP1            
Segment Reporting [Line Items]            
Operating expenses [1] 1,073,000   818,000   1,818,000 1,160,000
MECP2            
Segment Reporting [Line Items]            
Operating expenses [1] 0   (200,000)   0 (53,000)
Other external program costs            
Segment Reporting [Line Items]            
Operating expenses 3,990,000   1,841,000   7,169,000 3,670,000
Payroll and other personnel costs - R&D            
Segment Reporting [Line Items]            
Operating expenses [2] 18,218,000   11,618,000   34,336,000 22,629,000
Payroll and other personnel costs - SG&A            
Segment Reporting [Line Items]            
Operating expenses [2] 13,318,000   8,979,000   24,375,000 17,908,000
Other non-program costs - R&D            
Segment Reporting [Line Items]            
Operating expenses [3] 1,628,000   1,615,000   3,308,000 3,022,000
Other non-program costs - SG&A            
Segment Reporting [Line Items]            
Operating expenses [3] $ 5,587,000   $ 3,317,000   $ 10,742,000 $ 8,914,000
[1] Includes external research and development and sales, general and administrative expenses associated with programs.
[2] Includes salaries, benefits and other personnel related expenses, including stock based compensation expense, of relevant personnel.
[3] Includes costs incurred for conducting non-program activities related to Company activities.