LUXTURNA (voretigene) · 2017 · The Lancet
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Subretinal AAV2-hRPE65 gene therapy improves functional vision in biallelic RPE65-associated inherited retinal dystrophy.
Source publication
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al. · The Lancet · 2017
Sponsor
Spark Therapeutics, Inc.
Principal investigator
Albert M Maguire, MD
Population
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis; n=31
Primary endpoint
Multi-luminance Mobility Testing (MLMT), Bilateral
Methodology notes
Full methodology is under curation. This project aggregates 3 seeded claims across three temporal layers. The replication spec, pre-registered analysis, and version history will appear here as the project matures.
Funding progress
$1.1M / $4.0M
27%
Funding displayed here is indicative of collaborator interest. Sciync does not broker money, grants, or securities.
Temporal layers
Published findings under validation
1
Active replication in progress
1
Forecast horizons pending resolution
1