The following table provides a comprehensive list of all clinical studies conducted and submitted in this application. The table includes study identifiers, phase, and a brief description of each study's contribution to the overall development program for voretigene neparvovec. | Study ID | Phase | Title/Description | Population | Contribution | |---|---|---|---|---| | AAV2-RPE65v2-101/102 | 1 | Open-Label, Dose-Escalation Safety Study | 12 Subjects | Established safety, dose selection, initial evidence of efficacy and durability. | | AAV2-RPE65v2-301/302 | 3 | Randomized, Controlled Efficacy and Safety Study | 31 Subjects | Pivotal evidence of efficacy and safety. | | MTVS-201 | N/A | Mobility Test Validation Study | 60 Subjects | Validated the novel MLMT primary endpoint. | | RPE65-NHS-001 | N/A | Natural History Study of RPE65-mediated IRD | 70 Subjects | Characterized disease progression and established unmet medical need. |