Approved gene therapies embrace Luxturna to treat RPE65-mediated inherited retinal dystrophy, Glybera to cure familial lipoprotein lipase (LPL) deficiency (LPLD), while Roctavian (BMN 270) promising as therapeutic for hemophilia A, is awaiting approval from the Food and Drug Administration. Here, RPE65 is linked to inherited retinal dystrophy.