3.1
Voretigene neparvovec (Luxturna; Novartis Pharmaceuticals UK) is an adeno-associated virus vector-based gene therapy. It introduces a healthy copy of the defective RPE65 gene into the retinal cells of people with RPE65-mediated inherited retinal dystrophy (IRD), enabling patients to produce functional RPE65 protein. The aim is to improve visual function (performance of the eyes) and functional vision (the ability to carry out activities of daily living dependent on vision). Voretigene neparvovec has a marketing authorisation for 'the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells'.