After extensive clinical development, in 2017, voretigene neparvovec (VN; AAV2-hRPE65v2) received the U.S. Food and Drug Administration (FDA) approval for pediatric patients with RPE65-associated Leber congenital amaurosis (LCA) and confirmed biallelic RPE65-mediated retinal dystrophy, which are conditions that result in progressive vision loss, making it the first-ever FDA-approved gene therapy.7 The gene discussed is RPE65; the disease is Leber congenital amaurosis.