Press Release

Audentes Therapeutics Announces Presentation of Data at the American Society of Gene and Cell Therapy Annual Meeting

SAN FRANCISCO, CA – April 27, 2016 – Audentes Therapeutics, Inc., a biotechnology company focused on developing and commercializing gene therapy products for patients with serious, rare diseases, announced today that data related to its product candidate AT132 will be presented at the 19th Annual Meeting of the American Society of Gene and Cell Therapy to be held in Washington, D.C. on May 4-7th, 2016. The promising results from a long-term pre-clinical study of AT132 in a dog model of X-linked Myotubular Myopathy (XLMTM) support the advancement of this novel therapeutic into clinical development.

Data from an ongoing study in the naturally occurring dog model of XLMTM confirm earlier observations demonstrating the durable effects of treatment with AT132. At more than three years following a single IV administration of AT132, the dogs in the study showed prolonged survival and near-normal levels of neurological function, strength, gait and respiratory function, as well as normal muscle pathology as compared with age-matched untreated XLMTM and unaffected controls. These data will be presented by Matthew Elverman, M.D. of the University of Washington School of Rehabilitation Medicine.

Details of the presentation are listed below:

Oral Abstract #498

Title:                        Elverman, et. al., Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy.

Session:                  Musculo-skeletal Diseases

Date/Time:            Friday May 6th, 2016 from 4:15 – 4:30 pm

Room:                     Maryland Suite

In addition, Audentes is proud to co-sponsor The Outstanding New Investigator Awards, to be presented on May 5th from 1:30 – 3:30 in Salon 1-3 and to sponsor the Outstanding Achievement Award Lecture, to be held on May 6th from 10:45 to 11:45 am in Salon 1-3.

About X-Linked Myotubular Myopathy (XLMTM)

X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness, respiratory impairment and early mortality. There is no approved treatment for the condition. It is caused by mutations in the MTM1 gene, which encodes a protein called myotubularin. Myotubularin plays an important role in the development, maintenance and function of muscle cells. XLMTM affects approximately 1 in 50,000 newborn males worldwide. Audentes is developing AT132 for the treatment of XLMTM in collaboration with Genethon ( AT132 is a novel product based on AAV gene therapy technology.

About Audentes Therapeutics, Inc.

Audentes Therapeutics is a biotechnology company focused on developing and commercializing gene therapy products for patients suffering from serious, life-threatening rare diseases. We have four products in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT) and AT982 for the treatment of Pompe disease. We are a focused, experienced, and passionate team committed to forging strong, global relationships with the patient, research, and medical communities.