RPE65 and retinitis pigmentosa 1: Retinitis pigmentosa (RP) is the most prevalent group of inherited retinal dystrophy (IRD) in the world, with an estimated incidence of 1 in 4000 persons.1 In recent years, significant advancement has been made in the field of IRD with the Food and Drug Administration approval of voretigene neparvovec (Luxturna) for the treatment of RPE65-mediated IRD.2 According to www.clinicaltrials.gov (accessed 16 May 2021), there are 39 active interventional clinical trials for RP that are currently recruiting or enrolling subjects, and an additional 10 active studies that are not yet recruiting.