Hemgenix (etranacogene dezaparvovec-drlb, CSL Behring LLC) was submitted to the FDA on 24 March 2022 and approved on 22 November 2022 for the treatment of hemophilia B. Luxturna (voretigene neparvovec-rzyl, Spark Therapeutics Inc.)was submitted to the FDA on 16 May 2017 and approved on 18 December 2017 for the treatment of biallelic RPE65 mutation-associated retinal dystrophy. The gene discussed is RPE65; the disease is Retinal dystrophy.