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v3.25.1

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.

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