The FDA (Food and Drug Administration de EEUU) has just approved the first gene therapy treatment for inherited retinal dystrophies that targets a rare disease caused by mutations in a specific gene (precision medicine). After successful clinical trials, FDA has granted LUXTURNA to Spark Therapeutics. With a cost around 850.000 $ (two eyes treatment), LUXTURNA is addressed to patients that carry biallelic mutations in the RPE65, a gene causative of Leber Congenital Amaurosis (LCA), an early-onset severe retinal disease that leads to blindness. The treatment for children and young adult patients consists of a one-time subretinal injection of an adenoassociated therapeutic virus that introduces a correct copy of the RPE65 gene to rescue retinal function, thereby halting disease progression. LUXTURNA significantly preserves vision by preventing photoreceptor loss. Currently, only in the US about 2000 patients bearing mutations in RPE65 would be amenable to LUXTURNA treatment.
Image of LUXTURNA (voretigene neparvovec-rzyl) product vial, from Spark Therapeutics website