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Voretigene neparvovec for Leber congenital amaurosis (RPE65) – Phase 3 bilateral subretinal delivery
2016-002109-20Phase 3
- Sponsor
- Spark Therapeutics
- Start
- Oct 2016
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 3, single-arm trial of voretigene neparvovec in Leber congenital amaurosis (LCA), sponsored by Spark Therapeutics. Primary endpoint: Mobility testing.
02
Drugs
voretigene neparvovec
03
Conditions
Leber congenital amaurosis (LCA)
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