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Segment Information - Summary of Segment Information (Details) - USD ($)
 3 Months Ended
Mar. 31, 2025
Mar. 31, 2024
Segment Reporting Information [Line Items]    
Revenues $ 158,569,000 $ 4,216,000
Subtotal (47,329,000) (32,588,000)
Interest expense (1,000) (22,000)
Interest income 2,890,000 2,448,000
Other income (expense) 28,000 (428,000)
Provision for income taxes 1,278,000 0
Total other segment items 1,639,000 1,998,000
Net income (loss) 112,879,000 (26,374,000)
Zorevunersen    
Segment Reporting Information [Line Items]    
Subtotal [1] 17,674,000 8,780,000
ADOA    
Segment Reporting Information [Line Items]    
Subtotal [1] 1,201,000 1,722,000
SYNGAP1    
Segment Reporting Information [Line Items]    
Subtotal [1] 342,000 284,000
MECP2    
Segment Reporting Information [Line Items]    
Subtotal [1] 147,000 242,000
Other external program costs    
Segment Reporting Information [Line Items]    
Subtotal 1,829,000 2,043,000
Payroll and other personnel costs - R&D    
Segment Reporting Information [Line Items]    
Subtotal [2] 11,012,000 8,163,000
Payroll and other personnel costs - G&A    
Segment Reporting Information [Line Items]    
Subtotal [2] 8,929,000 6,850,000
Other non-program costs - R&D    
Segment Reporting Information [Line Items]    
Subtotal [3] 1,407,000 1,460,000
Other non-program costs - G&A    
Segment Reporting Information [Line Items]    
Subtotal [3] $ 4,788,000 $ 3,044,000
[1] Includes external research and development and 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.