RPE65 and Abnormal retinal morphology: During the past years, academic and pharmaceutical research activities in the field of ocular gene therapy have been intensified and led to marketing authorization of voretigene neparvovec, the first-in-class US Food and Drug Administration–approved gene therapy for inherited retinal diseases caused by variants in the retinal pigment epithelium–expressed gene RPE65. Multiple other forms of inherited blindness still remain without treatment options.