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Audentes Therapeutics Announces Continued Expansion Of Senior Management Team

John T. Gray, Ph.D., Brings Leading Gene Therapy Research and Manufacturing Expertise to Company

SAN FRANCISCO, CA – October 1, 2014 – Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, has announced the continued expansion of its leadership team, including the appointment of John T. Gray, PhD., as Vice President, Research and Development.  Dr. Gray brings more than 20 years of gene therapy research and manufacturing experience to Audentes.  In addition to Dr. Gray, Audentes has appointed Mary Newman as Senior Vice President, Regulatory Affairs and Dawn Blessing as Vice President, Corporate Development.

“We are thrilled to add three more experienced and passionate leaders to the Audentes team,” said Matthew Patterson, President and Chief Executive Officer of Audentes.  “Each will play a critical role in the advancement of our current programs into clinical trials next year and the growth of the company for the future. Dr. Gray’s expertise in the science and production of gene therapy products will be particularly valuable as Audentes advances in this rapidly evolving area of scientific innovation.”

John T. Gray joins Audentes with over 20 years of experience designing genetic therapies and vaccines, and developing manufacturing processes for those products.  For 11 years prior to joining Audentes, Dr. Gray was the Director of Vector Production and Development at St. Jude Children’s Research Hospital where he led a team devoted to advancing the gene therapy vector science.  In the area of lentiviral vector production, his team derived the GPRG stable cell line, the first such line to be used to successfully produce an HIV-based vector tested in a human clinical trial (for treatment of X-linked Severe Combined Immunodeficiency).  He also contributed significantly to the Hemophilia B gene therapy project, for which he designed the self-complementary AAV Factor IX vector expression cassette and developed the production process used to manufacture the first two batches of clinical vector.  During his tenure at St. Jude, which began in 2003, Dr. Gray also worked on Chimeric Antigen Receptor modified cell therapy, lysosomal storage disorder gene therapy, and multiple hematopoietic stem/progenitor cell gene therapy projects. Prior to joining St. Jude, Dr. Gray served as the assistant director of the Harvard Gene Therapy Initiative and prior to that, worked at Pfizer Animal Health designing bacterial and viral vectors for vaccine applications.  Dr. Gray has a Bachelor of Arts degree in Biochemistry from the University of California, Berkeley, and a Ph.D. degree in Biochemistry from the University of Colorado, Boulder.

Ms. Newman brings over 25 years of experience in regulatory affairs, and research and development, within the biotechnology industry. She joins Audentes after serving 7 years at SARcode Inc. (acquired by Shire Ltd.), most recently as Senior Vice President, Regulatory Affairs and Quality Assurance. Ms. Newman’s responsibilities included oversight of global regulatory strategic development, all primary regulatory agency interactions, and quality assurance for SARcode products. She previously held various management positions, with increasing responsibilities in Regulatory Affairs, at BioMarin Pharmaceutical, Inc., Berlex Inc. (now Bayer HealthCare Pharmaceuticals. Inc.), and Sequus Pharmaceuticals, Inc. (now Johnson and Johnson). Also at BioMarin, Ms. Newman oversaw the development and approval of Kuvan (sapropterin HCl) for the treatment of phenylketonuria (PKU), Naglazyme (galsulfase) for mucopolysaccharidosis (MPS) VI, and supported the final approval of Aldurazyme (laronidase) for MPS I. She has also held various leadership roles in research and development in oncology, neurology, and antifungal therapeutic areas. Ms. Newman earned Bachelor of Science and Master of Science degrees from Oregon State University.

Ms. Blessing joins Audentes with more than 18 years of experience in biotechnology finance, business development, and alliance management. Over this period, Ms. Blessing focused on programs for rare diseases and the application of genetic information to drug development. Ms. Blessing served as Senior Director, Business Development and Alliance Management at 23andMe Inc., a personal genetics company. In her role at 23andMe, she led multiple genomic research programs with partners in the pharmaceutical industry. Prior to 23andMe, Ms. Blessing spent 8 years in Business and Corporate Development at BioMarin Pharmaceutical Inc., originating or leading successful licensing efforts in multiple therapeutics areas for rare diseases, including preclinical and clinical-stage products. Prior to her role in industry, Ms. Blessing was a Biotechnology Equity Research analyst at Cowen and Company LLC, UBS Securities LLC, and Needham and Company LLC. Ms. Blessing earned a Bachelor of Science in Chemistry from the University of Richmond and a Masters of Business Administration from Columbia Business School.

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.