Summary of Significant Accounting Policies (Tables)
|
12 Months Ended |
Dec. 31, 2025 |
| Summary of Significant Accounting Policies |
|
| Schedule of Restricted Cash |
| | | | | | | | | December 31, | | December 31, | | | 2025 | | 2024 | Cash and cash equivalents | | $ | 65,931 | | $ | 103,659 | Restricted cash | | | 2,262 | | | 2,009 | Total cash, cash equivalents and restricted cash in the consolidated statement of cash flows | | $ | 68,193 | | $ | 105,668 |
|
| Schedule of Financial Assets and Liabilities Measured at Fair Value |
| | | | | | | | | | | | | | | Fair Value Measurement Using: | | | | | | Significant | | Significant Other | | Significant | | | December 31, | | Observable Inputs | | Observable Inputs | | Unobservable | Description | | 2025 | | (Level 1) | | (Level 2) | | (Level 3) | Cash equivalents | | $ | 60,253 | | $ | 60,253 | | $ | — | | $ | — | Restricted cash | | $ | 2,262 | | $ | 2,262 | | $ | — | | $ | — |
| | | | | | | | | | | | | | | Fair Value Measurement Using: | | | | | | Significant | | Significant Other | | Significant | | | December 31, | | Observable Inputs | | Observable Inputs | | Unobservable | Description | | 2024 | | (Level 1) | | (Level 2) | | (Level 3) | Cash equivalents | | $ | 80,930 | | $ | 80,930 | | $ | — | | $ | — | Restricted cash | | $ | 2,009 | | $ | 2,009 | | $ | — | | $ | — |
|
| Schedule of Estimated Useful Lives of Assets |
| | | Asset Category | | Useful Lives | Computer and office equipment | | 3 years | Laboratory equipment | | 5 years | Furniture and fixtures | | 5 years | Manufacturing equipment | | 7 years | Leasehold improvements | | lesser of useful life or remaining term of lease |
|
| Schedule of Change in Asset Retirement Obligations |
| | | | | | | | | For the Years Ended December 31, | | | 2025 | | 2024 | Balance at beginning of period | | $ | 2,821 | | $ | 2,401 | Change in estimate | | | (1,676) | | | 345 | Assignment of leases to Johnson & Johnson Innovative Medicine | | | — | | | (115) | Amortization of interest | | | 153 | | | 209 | Effects of exchange rate changes | | | 101 | | | (19) | Balance at end of period | | $ | 1,399 | | $ | 2,821 |
|
| Schedule of Securities Excluded from Computation of Diluted Net Loss per Share |
| | | | | | | December 31, | | December 31, | | | 2025 | | 2024 | Share options | | 7,859,338 | | 8,232,587 | Restricted share units | | 6,184,250 | | 4,252,250 | Warrants | | 700,000 | | 700,000 | | | 14,743,588 | | 13,184,837 |
|
| Schedule of Net Loss for the Segment |
| | | | | | | | | For the Years Ended December 31, | | | 2025 | | 2024 | Service revenue - related party | | $ | 6,400 | | $ | 33,279 | License revenue | | | 74,991 | | | — | Operating expenses: | | | | | | | Cost of service revenue - related party | | | 4,843 | | | 23,791 | General and administrative | | | 38,480 | | | 39,389 | Clinical programs: | | | | | | | Botaretigene sparoparvovec | | | — | | | 1,250 | AAV-hAQP1 | | | 13,762 | | | 17,307 | AAV-CNGB3 / AAV-CNGA3 | | | — | | | (969) | AAV-GAD | | | 14,268 | | | 6,411 | Other ocular diseases | | | 4,380 | | | 1,763 | Manufacturing | | | 44,288 | | | 53,445 | Preclinical programs: | | | | | | | Gene regulation | | | 9,426 | | | 10,509 | Neurodegenerative diseases | | | 1,116 | | | 1,608 | Preclinical ocular diseases | | | 3,567 | | | 2,474 | Other research and development1 | | | 18,582 | | | 2,494 | Share-based compensation | | | 22,105 | | | 25,191 | Depreciation and amortization | | | 12,542 | | | 12,828 | Total operating expenses | | | 187,359 | | | 197,491 | Other segment items2 | | | (8,233) | | | 16,421 | Segment net loss | | $ | (114,201) | | | (147,791) | Net loss | | $ | (114,201) | | $ | (147,791) |
1 Other research and development is comprised of all other costs including payroll and payroll related costs, travel, rent and facilities costs and other non-program specific expenses. 2 Other segment items is comprised of foreign currency gain (loss), interest income, interest expense and gain on sale of nonfinancial assets.
|
| Schedule of Long-lived Assets by Geographical Area |
| | | | | | | | | December 31, | | December 31, | | | 2025 | | 2024 | United States | | $ | 8,830 | | $ | 7,209 | United Kingdom | | | 26,092 | | | 28,419 | European Union | | | 107,589 | | | 100,845 | | | $ | 142,511 | | $ | 136,473 |
|