Priority Review for Spark's ophthalmic gene therapy

Spark Therapeutics Inc. (NASDAQ:ONCE) said FDA accepted and granted Priority Review to a BLA for gene therapy Luxturna voretigene neparvovec (AAV2-hRPE65v2) to treat vision loss due to confirmed biallelic retinal pigment epithelium-specific protein 65kDa (RPE65)-mediated inherited retinal disease (IRD). Its PDUFA date is Jan. 12, 2018.

Spark said the therapy, which consists of an adeno-associated virus (AAV) encoding the RPE65 gene, could be the first gene therapy approved in the U.S., as well as the first pharmacologic treatment for an IRD. ...