Spark outlines 2018 goals

CEO Jeffrey Marrazzo said Spark Therapeutics Inc. (NASDAQ:ONCE) expects to have 10 clinical-stage gene therapy programs in the clinic by 2018, with data readouts from five programs expected by 1H17. The details came at a presentation at the JPMorgan Healthcare Conference in San Francisco late Monday.

Of the 10 programs, Marrazzo said Spark hopes to have one approved product: voretigene neparvovec ( SPK-RPE65) to treat retinal pigment epithelium-specific protein 65kDa (RPE65)-mediated inherited retinal dystrophies (IRDs). Spark plans to submit a BLA to FDA next half and an MAA to EMA in early 2017 for the adeno-associated virus (AAV) carrying the RPE65 gene. ...