Among these clinical trials, voretigene neparvovec (Luxturna) that targets RPE65-associated LCA was the first FDA-approved gene therapy drug, while other clinical trials of IRDs, including Retinitis Pigmentosa (RP), Choroideremia and X-linked Retinitis Pigmentosa (XLRP), are also under way. The gene discussed is RPE65; the disease is Leber congenital amaurosis.