Spark Therapeutics is poised to bring the first gene therapy for an inherited disorder to market in the United States. In May, the company completed its rolling biologics license application submission to the US Food and Drug Administration (FDA) for voretigene neparvovec, a gene therapy for RPE65-mediated inherited retinal disease, a rare retinal dystrophy that causes blindness.
Abbasi J. Spark Advances Gene Therapy for Inherited Vision Loss. JAMA. 2017;318(2):116. doi:10.1001/jama.2017.8767