RPE65 and achromatopsia: The feasibility of using gene therapy safely to successfully treat inherited eye disease was demonstrated recently with the first FDA- and EMA-approved gene therapy for RPE65-associated retinal dystrophy, Leber’s congenital amaurosis, a severe early-onset blinding disease.8 There are currently three phase I/II gene therapy trials for CNGA3-associated ACHM (NCT03758404, NCT02935517 and NCT02610582), and two phase I/II gene therapy trials for CNGB3-ACHM (NCT03001310 and NCT02599922).