v3.26.1
Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Net Income (loss) for the Segment (Details)
$ in Thousands
 3 Months Ended 6 Months Ended
Jun. 30, 2026
USD ($)
Jun. 30, 2025
USD ($)
Jun. 30, 2026
USD ($)
Jun. 30, 2025
USD ($)
Operating expenses:        
Number of reportable segments     1  
Number of operating segments     1  
Revenues $ 321,434 $ 3,691 $ 321,727 $ 5,617
Share-based compensation     7,433 10,551
Depreciation and amortization     5,831 6,271
Total operating expenses 77,009 48,484 118,119 92,006
Net Income (Loss) 160,720 (38,795) 114,407 (78,776)
Non-related party        
Operating expenses:        
Cost of service revenue 1,401   1,401  
Related party        
Operating expenses:        
Cost of service revenue 5,763 2,676 5,961 4,054
Service revenue - related party        
Operating expenses:        
Revenues 104,906 3,691 105,199 5,617
Service revenue        
Operating expenses:        
Revenues 11,885   11,885  
License revenue - related party        
Operating expenses:        
Revenues 204,643   204,643  
Single reportable segment        
Operating expenses:        
General and administrative 9,146 8,607 15,316 14,951
Botaretigene sparoparvovec 31,302   31,331  
AAV-hAQP1 7,349 2,754 9,704 7,606
AAV-GAD 3,098 8,970 4,010 10,430
Other ocular diseases   20 108 1,465
Manufacturing 5,046 8,888 21,409 22,989
Gene regulation 1,252 3,241 2,673 5,527
Neurodegenerative diseases 328 298 729 724
Preclinical ocular diseases 180 1,162 1,003 1,787
Other research and development 5,540 3,019 11,210 5,651
Share-based compensation 3,779 5,659 7,433 10,551
Depreciation and amortization 2,825 3,190 5,831 6,271
Total operating expenses 77,009 48,484 118,119 92,006
Other segment items (83,756) 5,998 (89,252) 7,613
Segment net income (loss) 160,669 (38,795) 114,356 (78,776)
Net Income (Loss) 160,669 (38,795) 114,356 (78,776)
Single reportable segment | Non-related party        
Operating expenses:        
Cost of service revenue 1,401   1,401  
Single reportable segment | Related party        
Operating expenses:        
Cost of service revenue 5,763 2,676 5,961 4,054
Single reportable segment | Service revenue - related party        
Operating expenses:        
Revenues 104,906 $ 3,691 105,199 $ 5,617
Single reportable segment | Service revenue        
Operating expenses:        
Revenues 11,885   11,885  
Single reportable segment | License revenue - related party        
Operating expenses:        
Revenues $ 204,643   $ 204,643