Audentes Therapeutics and Genethon Announce Agreement to Develop Treatment for Severe Genetic Disease X‐Linked Myotubular Myopathy

SAN FRANCISCO, CA – February 5, 2014 –Audentes Therapeutics, Inc., a biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, and Genethon, a non‐profit organization dedicated to the research and development of biotherapies for orphan genetic diseases, announce that they have entered into an agreement to develop AT001 for the treatment of X‐Linked Myotubular Myopathy (XLMTM), a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. AT001 is a novel drug candidate based on adeno‐associated virus (AAV) gene therapy technology.