Product
Voretigene neparvovec
2 clinical trials
2 indications
Indication
Leber congenital amaurosisClinical trial
An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal DystrophyStatus: Active (not recruiting), Estimated PCD: 2022-04-05
Clinical trial
A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 StudyStatus: Active (not recruiting), Estimated PCD: 2030-03-01