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September 01, 2015 3:31 PM ET


Company Overview of Audentes Therapeutics, Inc.

Company Overview

Audentes Therapeutics, Inc., a biotechnology company, engages in the development and commercialization of new treatments for people with serious rare muscle diseases through the application of adeno-associated virus gene therapy technology. Its treatments include AT001 for the treatment of X-linked myotubular myopathy; and AT002 for the treatment of pompe disease. Audentes Therapeutics, Inc. was founded in 2012 and is based in San Francisco, California.

San Francisco, CA 

United States

Founded in 2012

Key Executives for Audentes Therapeutics, Inc.

Chief Executive Officer, President and Director
Age: 43
Chief Medical Officer and Senior Vice President
Senior Vice President of Preclinical Development and Translational Medicine
Senior Vice President of Regulatory Affairs and Quality Assurance
Vice President of Corporate Development
Compensation as of Fiscal Year 2015.

Audentes Therapeutics, Inc. Key Developments

Audentes Therapeutics Receives Orphan Drug Designation for AT001 for the Treatment of X-Linked Myotubular Myopathy in the US and Europe

Audentes Therapeutics, Inc. announced that AT001, an investigational product in development for the treatment of X-Linked Myotubular Myopathy (XLMTM), has been granted orphan designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). XLMTM is a rare, severe, inherited disorder that affects skeletal muscles from birth. The disease is caused by mutations in the MTM1 gene, which encodes an enzyme called myotubularin. Myotubularin plays an important role in the development, maintenance and function of muscle cells. AT001 is a novel product based on adeno-associated virus (AAV) gene therapy technology that is designed to restore the ability of skeletal muscle to produce myotubularin. Administration of AT001 has resulted in improved muscle strength and survival in a murine model of XLMTM as well as improved muscle strength, respiratory symptoms and survival in a canine model of the disease. The FDA and EMA Orphan Drug programs provide orphan designation to drugs and biologics that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States or 5 in 10,000 in the EU). The designation provides sponsors with development and commercial incentives including, seven years of market exclusivity in the US, 10 years of market exclusivity in the EU, consultation by FDA and EMA on clinical study design, and certain fee exemptions and reductions.

Audentes Therapeutics, Inc. Presents at Piper Jaffray GenomeRx Symposium, May-20-2015

Audentes Therapeutics, Inc. Presents at Piper Jaffray GenomeRx Symposium, May-20-2015 . Venue: The New York Palace Hotel, New York, New York, United States. Speakers: John T. Gray, Vice President of Research and Development, Matthew R. Patterson, Chief Executive Officer, President and Director.

Audentes Therapeutics Appoints Stephen Squinto to its Board of Directors

Audentes Therapeutics, Inc. 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. 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.

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