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Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Tables) | 3 Months Ended
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Mar. 31, 2026
Summary of Significant Accounting Policies and Recent Accounting Pronouncements |
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
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 | ​ | $ | — | ​ | $ | —
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
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
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
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.
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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