Efficacy and Safety of Voretigene Neparvovec in

Purpose: We report the efficacy and safety of voretigene neparvovec (VN) as an adeno-associated viral vector-based gene therapy for Japanese patients with inherited retinal dystrophy caused by biallelic pathogenic Design: Open-label, single arm, phase III clinical trial. Participants: Four subjects were recruited based on the following criteria: (1) a clinical and molecular genetic diagnosis of Methods: All subjects received subretinal injections of VN (1.5 × 10 Main Outcome Measures: The primary efficacy end point was a change from baseline in full-field light sensitivity threshold (FST) (white light, averaged over both eyes) at days 30, 90, 180, 270, and year 1. The secondary efficacy end points included changes from baseline in VF testing by Goldmann kinetic perimetry (GP) and BCVA in the logarithm of the minimum angle of resolution (LogMAR) unit. Safety was evaluated by adverse events (AEs), laboratory evaluations, and opthalmic examinations. Results: The mean baseline age of 4 subjects was 31.3 years (10, 17, 49, and 49 years). The homozygous pathogenic variants (c.1543C>T, p.Arg515Trp) were identified in 3 subjects. The mean (range) FST averaged over both eyes improved by -1.831 (-3.54 to -0.56) log Conclusions: Overall, these data from a phase III trial showed improvements in FST and VF and well-tolerated safety profiles of VN for 1 year, with ongoing follow-up of up to 5 years in Japanese patients with Financial Disclosures: Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.