Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Tables)
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3 Months Ended |
Mar. 31, 2026 |
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| Summary of Significant Accounting Policies and Recent Accounting Pronouncements |
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| Schedule of Restricted Cash |
| | | | | | | | | March 31, | | December 31, | | | 2026 | | 2025 | Cash and cash equivalents | | $ | 71,541 | | $ | 65,931 | Restricted cash | | | 2,217 | | | 2,262 | Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows | | $ | 73,758 | | $ | 68,193 |
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| Schedule of Financial Assets and Liabilities Measured at Fair Value |
| | | | | | | | | | | | | | | Fair Value Measurement Using: | | | | | | Significant | | Significant Other | | Significant | | | March 31, | | Observable Inputs | | Observable Inputs | | Unobservable | Description | | 2026 | | (Level 1) | | (Level 2) | | (Level 3) | Cash equivalents | | $ | 13,929 | | $ | 13,929 | | $ | — | | $ | — | | | | | | | | | | | | | |
| | | | | | | | | | | | | | | 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 | | $ | — | | $ | — | | | | | | | | | | | | | |
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| Schedule of Change in Asset Retirement Obligations |
| | | | | | | | | For the Three-Month Periods Ended March 31, | | | 2026 | | 2025 | Balance at beginning of period | | $ | 1,399 | | $ | 2,821 | Change in estimate | | | — | | | (1,576) | Amortization of interest | | | 36 | | | 63 | Effects of exchange rate changes | | | (24) | | | 36 | Balance at end of period | | $ | 1,411 | | $ | 1,344 |
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| Schedule of Securities Excluded from Computation of Diluted Net Loss per Share |
| | | | | | | March 31, | | March 31, | | | 2026 | | 2025 | Share options | | 7,548,411 | | 8,022,989 | Restricted share units | | 4,570,000 | | 3,389,750 | Warrants | | 700,000 | | 700,000 | | | 12,818,411 | | 12,112,739 |
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| Schedule of Net Loss for the Segment |
| | | | | | | | | | For the Three-Month Periods Ended March 31, | | | | 2026 | | 2025 | | Service revenue - related party | | $ | 293 | | $ | 1,926 | | Operating expenses: | | | | | | | | Cost of service revenue - related party | | | 198 | | | 1,378 | | General and administrative | | | 6,158 | | | 6,348 | | Clinical programs: | | | | | | | | AAV-hAQP1 | | | 2,353 | | | 4,852 | | AAV-GAD | | | 847 | | | 1,459 | | Other ocular diseases | | | — | | | 1,445 | | Manufacturing | | | 16,422 | | | 17,359 | | Preclinical programs: | | | | | | | | Gene regulation | | | 1,366 | | | 2,286 | | Neurodegenerative diseases | | | 297 | | | 426 | | Ocular diseases | | | 1,145 | | | 625 | | Other research and development1 | | | 5,662 | | | (624) | | Share-based compensation | | | 3,654 | | | 4,887 | | Depreciation and amortization | | | 3,008 | | | 3,081 | | Total operating expenses | | | 41,110 | | | 43,522 | | Other segment items2 | | | (5,496) | | | 1,615 | | Segment net loss | | $ | (46,313) | | $ | (39,981) | | Net loss | | $ | (46,313) | | $ | (39,981) | |
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, and interest expense.
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| Schedule of Long-lived Assets by Geographical Area |
The Company’s service revenue – related party and deferred revenue – related party from the Original Asset Purchase Agreement and related agreements were generated in the United Kingdom. The following table summarizes long-lived assets by geographical area (in thousands): | | | | | | | | | March 31, | | December 31, | | | 2026 | | 2025 | United States | | $ | 8,426 | | $ | 8,830 | United Kingdom | | | 25,991 | | | 26,092 | European Union | | | 103,605 | | | 107,589 | | | $ | 138,022 | | $ | 142,511 |
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