FDA approved Luxturna, a gene therapy product for inherited blindness

The FDA has approved Luxturna for clinical use in the US. The product, marketed by Spark Therapeutics, is a treatment for a form of inherited blindness called RPE65-mediated inherited retinal dystrophy. Luxturna is an AAV vector carrying a healthy copy of the RPE65 gene. It is administered via one-time injection directly into the eye. Clinical trials that were published earlier this year in The Lancet showed improved functional vision after 1 year without serious adverse effects. The approval follows approvals for two CAR T cell treatments in August (Kymriah by Novartis) and October (Gilead’s Yescarta), making 2017 a highly successful year for gene and cell therapies.