The Phase 3 trial “A Phase III Trial of Gene Therapy for LCA Due to RPE65 Mutations” (ClinicalTrials.gov ID: NCT00999609), sponsored by Spark Therapeutics, Inc., is a multicenter, open-label, randomized controlled trial assessing the efficacy and safety of voretigene neparvovec-rzyl in patients with confirmed biallelic RPE65 mutation-associated IRD. The gene discussed is RPE65; the disease is Leber congenital amaurosis.