Spark Therapeutics enters into a licensing agreement with the big pharmaceutical company Novartis to commercialize LUXTURNA, the first gene therapy treatment approved for inherited retinal dystrophies, in Europe. LUXTURNA is addressed to patients suffering from Leber Congenital Amaurosis (LCA) caused by mutations in the RPE65 gene. This one-time gene therapy consists of a subretinal microinjection of therapeutic adenoassociated virus that contain a normal copy of RPE65. Corrected retinal pigment epithelium cells halt retinal degeneration and subsequent vision loss. LCA, an early onset severe disease that eventually leads to complete blindnes in pediatric and young adult patients, had no pharmacological treatment options. The license agreement involved an ex-ante payment of more than 100 milions dollars by Novartis.
Spark has licensed Novartis for commercialization of LUXTURNA, the first approved gene therapy treatment for a rare form of inherited blindness, outside the US.