2:47 PM
 | 
Jan 09, 2018
 |  BC Extra  |  Company News

Spark to develop gene therapy 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...

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