Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Inclusion Criteria: * Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis designated laboratory in Japan. * Age four years or older. * Visual acuity worse than 20/60 (both eyes) and/or visual field less than 20 degrees in any meridian as measured by a III4e isopter or equivalent (both eyes). * Sufficient viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/ or ophthalmoscopy. Must have either: * An area of retina within the posterior pole of \> 100 µm thickness shown on OCT, or * ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole, or * Remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent Exclusion Criteria: * Any prior participation in a study in which a gene therapy vector was administered. * Participation in a clinical study with an investigational drug in the past 6 months from screening visit. * Known hypersensitivity to any of the study treatments including excipients or to medications planned for use in the peri-operative period. * Unable to reliably perform the FST assessment. * Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme in the past 6 months from screening visit. * Prior intraocular surgery within 6 months from screening visit. * Prior use of any…