Spark unveils gene therapy program for Pompe's

Spark Therapeutics Inc. (NASDAQ:ONCE) said it is developing a preclinical gene therapy for Pompe's disease using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA) transgene in-licensed last year from Genethon (Evry, France). Spark unveiled the program on Monday at the J.P. Morgan Healthcare Conference in San Francisco.

The company said the GAA transgene may overcome immunogenicity effects that reduce the efficacy of existing recombinant GAA used in enzyme replacement therapies. SPK-GAA is designed to enable continuous secretion of transgene-derived GAA from the liver...