Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Participants: ≥3yo and in each eye:
BCVA 20/50 or worse, visual field < 20 degrees in any meridian, with genetic diagnosis of biallelic RPE65 mutations

• n=31

Intervention:

Group 1: voretigene neparvovec (subretinal injection of 1.5×1011 in 0.3mL)
Group 2: Placebo

Mean bilateral multi-luminance mobility testing change score was significantly higher in the voretigene neparvovec group than in the control group (1.8 vs 0.2).

There were no product-related serious adverse events or deleterious immune responses.