Spark submission brings first gene therapy for genetic disease closer

US biotech firm Spark Therapeutics (Nasdaq: ONCE) has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Luxturna, the proposed trade name for voretigene neparvovec, an investigational, one-time gene therapy for patients with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations.

The MAA includes data from three clinical trials that enrolled 41 participants with RPE65-mediated inherited retinal dystrophy (IRD), including the first randomized, controlled Phase III trial for a gene therapy for a genetic disease.

"These individuals, who eventually will progress to complete blindness, have hope for a potential treatment option that may restore their vision"