Audentes Therapeutics, Inc., a recently founded biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of a $30 million Series A financing. The financing was led by OrbiMed Advisors with the participation of 5AM Ventures and Versant Ventures. This funding will allow the Company to further advance its two lead programs, AT001 for X-linked Myotubular Myopathy (XLMTM) and AT002 for Pompe disease, and to evaluate additional candidate programs. Both AT001 and AT002 are innovative, novel treatments based on adeno-associated virus (AAV) gene therapy technology.

'This financing is a tremendous step forward for the development of new potential treatments for patients affected by very serious orphan muscle diseases. I'm delighted to have the opportunity to work with such a fantastic group of people who share our vision for success in bringing these therapies to patients,' said Matthew R. Patterson, President and Chief Executive Officer of Audentes. 'Our focused, experienced team is committed to advancing the promising scientific approach of gene therapy and looks forward to collaborating closely with our patient, research and medical community partners in this process.'

Audentes, headquartered in San Francisco, CA, was founded by Mr. Patterson in November, 2012 in collaboration with Thomas J. Schuetz, M.D., Ph.D. and OrbiMed Advisors. The Audentes Board of Directors will consist of Jonathan Silverstein (OrbiMed Advisors), who will serve as Chairman, Kush Parmar, M.D., Ph.D. (5AM Ventures), Thomas Woiwode, Ph.D. (Versant Ventures), Dr. Schuetz, who will act as an independent director, and Mr. Patterson.

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 is thought to be involved in the development and maintenance of muscle cells. XLMTM affects approximately 1 in 50,000 newborn males worldwide.

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.

About Audentes Therapeutics, Inc.

AudentesTM is a biotechnology company committed to the development and commercialization of innovative new treatments for people with serious, rare muscle diseases through the application of adeno-associated virus (AAV) gene therapy technology. 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.