Press Release

Audentes Therapeutics Appoints Stephen Squinto, Ph.d. To Its Board Of Directors

SAN FRANCISCO, CA – April 21, 2015 – Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced the appointment of Stephen Squinto, Ph.D., to the company’s Board of Directors. Dr. Squinto, a co-founder of Alexion Pharmaceuticals, Inc., will serve as an independent director to Audentes.

“Steve is a pioneer in bringing innovative therapies to patients with rare diseases. As both a scientist and a business leader, his guidance will prove invaluable as we advance our lead programs into the clinic and continue to build for the future,” said Matthew Patterson, President and Chief Executive Officer of Audentes Therapeutics. “On behalf of the rest of the Board of Directors and the management team, I welcome Steve to Audentes.”

“Audentes is an exciting company that is poised to bring highly innovative treatments to patients and to further establish itself as a leader in the gene therapy field,” commented Stephen Squinto, Ph.D. “I am pleased to join the board and look forward to helping the team achieve its remarkable vision for success.”

Stephen Squinto, Ph.D., has over 25 years of experience in the biotechnology industry, as both a scientist and senior executive. He is currently a Venture Partner with OrbiMed. Dr. Squinto was a co-founder of Alexion Pharmaceuticals Inc. and recently served as its Executive Vice President and Chief Global Operations Officer. Prior to 2013, he was Alexion’s Global Head of Research and Development. Alexion is dedicated to developing therapies for certain rare and ultra-rare diseases. From 1988 to 1992, Dr. Squinto held various positions at Regeneron Pharmaceuticals, Inc. Prior to Regeneron, he held a joint academic position at both the Tulane University and LSU Medical Schools. He is a recipient of numerous honors and awards from academic and professional organizations for his scientific work. Dr. Squinto received his B.A. in Chemistry and Ph.D. in Biochemistry and Biophysics from Loyola University of Chicago.

About Audentes Therapeutics, Inc.
Audentes is a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases. The company has two products in development, AT001 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and AT002 for the treatment of Pompe Disease. The company consists of a focused, experienced, and passionate team driven by the goal of improving the lives of patients. Audentes takes pride in strong, global relationships with the patient, research, and medical communities.

For more information regarding Audentes, please visit www.audentestx.com.

About X-Linked Myotubular Myopathy (XLMTM)
X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. It is caused by mutations in the MTM1 gene, which encodes an enzyme called myotubularin. Myotubularin plays an important role in the development and maintenance of muscle cells. XLMTM affects approximately 1 in 50,000 newborn males worldwide. Audentes is developing AT001 for the treatment of XLMTM in collaboration with Genethon (www.genethon.fr). AT001 is a novel product based on AAV gene therapy technology.

About Pompe Disease
Pompe Disease is a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It is caused by mutations in a gene that encodes an enzyme called acid alpha-glucosidase (GAA), which is needed by the body to break down glycogen – a stored form of sugar used for energy. Pompe Disease affects approximately 1 in every 40,000 births. AT002 is a novel product based on AAV gene therapy technology.