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Revenue recognition - Collaboration and Royalty Revenue (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | 9 Months Ended |  |
--- | --- | --- | --- | --- | --- | ---
Jan. 31, 2025 USD ($) | Sep. 30, 2025 USD ($) | Sep. 30, 2024 USD ($) | Jun. 30, 2024 USD ($) | Jun. 30, 2024 USD ($) | Sep. 30, 2025 USD ($) product | Sep. 30, 2024 USD ($) | Dec. 31, 2024 USD ($) | Nov. 30, 2011 USD ($)
Revenue recognition |  |  |  |  |  |  |  |  |
Number of approved products | product |  |  |  |  |  | 2 |  |  |
Remaining potential milestones that can be achieved |  |  |  |  |  |  |  | $ 150,000 |
Revenue |  | $ 211,007 | $ 196,786 |  |  | $ 1,565,978 | $ 593,608 |  |
Deferred revenue |  | 1,691 |  |  |  | 1,691 |  | $ 5,505 |
Collaboration and license revenue |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Revenue |  | 9,258 |  |  |  | 998,430 |  |  |
Royalty revenue |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Revenue |  | 70,793 | 61,365 |  |  | $ 164,837 | 145,702 |  |
SMA License Agreement |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Number of approved products | product |  |  |  |  |  | 1 |  |  |
SMA License Agreement | Collaboration and license revenue |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Revenue |  |  |  | $ 0 | $ 0 |  |  |  |
SMA License Agreement | Royalty revenue |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Revenue |  | 70,800 | $ 61,400 |  |  | $ 164,800 | $ 145,700 |  |
License and Collaboration Agreement |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Refund of revenue related to prior collaboration arrangements |  |  |  |  |  | 3,500 |  |  |
License and Collaboration Agreement | Novartis |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Cash proceeds from upfront payment on collaboration agreement | $ 1,000,000 |  |  |  |  |  |  |  |
Percentage share of the income (loss) generated on U.S. sales | 40.00% |  |  |  |  |  |  |  |
Deferred revenue |  | 0 |  |  |  | 0 |  |  |
License and Collaboration Agreement | Novartis | Collaboration and license revenue |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Revenue |  | 9,300 |  |  |  | 1,000,000 |  |  |
Research And Development Event Milestones | SMA License Agreement |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Remaining potential milestones that can be achieved |  | 0 |  |  |  | 0 |  |  |
Sales Milestones | SMA License Agreement |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Remaining potential milestones that can be achieved |  | $ 150,000 |  |  |  | $ 150,000 |  |  |
Maximum | License and Collaboration Agreement | Novartis |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Potential future proceeds contingent on achievement of developmental, regulatory and sales milestones and tiered royalties | $ 1,900,000 |  |  |  |  |  |  |  |
Maximum | Research And Development Event Milestones | SMA License Agreement |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Remaining potential milestones that can be achieved |  |  |  |  |  |  |  |  | $ 135,000
Maximum | Sales Milestones | SMA License Agreement |  |  |  |  |  |  |  |  |
Revenue recognition |  |  |  |  |  |  |  |  |
Remaining potential milestones that can be achieved |  |  |  |  |  |  |  |  | $ 325,000

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