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. · PI Albert M Maguire, MD

Primary endpoint

Multi-luminance Mobility Testing (MLMT), Bilateral

Population

Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis; n=31

doi:10.1016/S0140-6736(17)31868-8

Historical

Current

Future

3 noemes

Historical

1 noeme

Published findings from this trial. Vote whether the expert body still believes each noeme stands given current evidence.

The LUXTURNA (voretigene) trial's stated primary conclusion — Subretinal AAV2-hRPE65 gene therapy improves functional vision in biallelic RPE65-associated inherited retinal dystrophy. — replicates in independent cohorts.

gene-therapy

Current

1 noeme

Noemes tracking active replication, subgroup analysis, or long-term follow-up. Vote on the likelihood the original effect holds.

Recent follow-up analyses of LUXTURNA (voretigene) are confirming the original effect size in real-world data.

gene-therapy

Future

1 noeme

Open forecast horizons tied to this trial. Vote your probability that each resolves TRUE by the declared horizon.

By 2028, at least two additional subretinal gene therapies (beyond LUXTURNA) will be FDA-approved for monogenic retinal dystrophies.

gene-therapy

71%

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