“One Step Closer to Clinical Trials!” Audentes Theraputics Moved Forward with Proposed Treatment for XLMTM

May 30, 2014

Three cheers for Audentes Theraputics, Inc. who this week announced the results of an ongoing preclinical study on a treatment for X-Linked Myotubular Myopathy (XLMTM.) XLMTM is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment.

Data will be presented at the 17th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), to be held in Washington D.C. May 21-24.

Previous testing of the study done on two animal models showed promising proof of concept for the potential treatment (AT001).

These animal studies demonstrated that treatment with a single dose of AAV carrying the gene deficient in XLMTM resulted in an increase in muscle strength, improved respiratory function, and prolonged survival.

New data from studies in the naturally occurring dog model of XLMTM, which will be presented at this year’s ASGCT, expand and confirm earlier observations. These data are the first demonstration of persistent disease correction in a large animal model of a neuromuscular disease through the delivery of a single, intravenous administration of AAV. The studies of AT001 have been performed in collaboration with Genethon and UW Medicine.

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 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

About Genethon

Genethon, located in Evry, France, is a non-profit R&D organization dedicated to the development of biotherapeutics for orphan genetic diseases, from research to clinical validation. Genethon specializes in the discovery and development of gene therapy drugs and has multiple ongoing programs at clinical, preclinical and research stages for neuromuscular, blood, immune system, liver, and eye diseases. To support clinical development of gene therapy drugs, Genethon has built one of the largest facilities worldwide for the production of clinical-grade gene therapy vectors.

For more information regarding Genethon, please visit

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