Audentes Therapeutics Announces Upcoming Presentations at 22nd Annual Meeting of the American Society of Gene and Cell Therapy Including New Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy

SAN FRANCISCO, April 15, 2019 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, today announced multiple planned presentations at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Washington, D.C., including new data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in patients with X-linked Myotubular Myopathy (XLMTM).  The oral presentation will be given by Perry B. Shieh, M.D., Ph.D., Associate Professor of Neurology at the Department of Neurology at UCLA Medical Center and will be held during the Plenary Session "Presidential Symposium & Presentation of the Top Abstracts," scheduled to begin at 2:15 pm ET. 

The presentation by Dr. Shieh will include new data from ASPIRO, with up to 48-weeks of follow-up in all patients enrolled in dose Cohort 1 (1x1014vg/kg), and up to 24-weeks in the earliest patients enrolled in dose Cohort 2 (3x1014 vg/kg).

"We remain highly encouraged by the emerging profile of AT132 and are looking forward to sharing new results from ASPIRO," stated Matthew R. Patterson, Chairman and Chief Executive Officer.  "2019 is a critical year for the development of AT132, with important next steps that include optimal dose selection and final agreement on regulatory submission pathways in the US and Europe, which we expect to achieve in the third quarter."

Audentes has a range of additional presentations planned during the conference, including a sponsored symposium that will provide an in-depth review of the new data from ASPIRO.  Following are details for each presentation:

Oral Presentations:

ASPIRO Phase 1/2 Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings
Abstract number: 652
Session Title: Presidential Symposium & Presentation of the Top Abstracts
Date: Wednesday, May 1, 2019, 2:15 pm - 2:30 pm ET

Combination Therapy Reduces Existing Anti-AAV Antibody by Logs and Allows for Safe and Efficacious Redosing
Abstract number: 147
Session Title: Use of Immune Modulation in Gene Therapy
Date: Monday, Apr 29, 2019, 4:00 pm - 4:15 pm ET

Development of an NGS-Based Assay and Bioinformatics Workflow for CMC Characterization of Contaminating DNA in AAV Products
Abstract number: 700
Session Title: Advances in Cell Product and Gene Vector Manufacturing
Date: Wednesday, May 1, 2019, 4:15 pm - 4:30 pm ET

Poster Presentation:

AAV9-U7snRNA Mediated Skipping of DMD Exon 2: Absence of Off-target Splicing Effects as Demonstrated by RNA-Seq
Abstract number: 814
Session Title: Musculo-skeletal Diseases
Date: Wednesday, May 1, 2019, 5:00 pm - 6:00 pm ET

Audentes-Sponsored Symposium:
Achieving New Milestones in Neuromuscular Disease: AAV Gene Therapy for X-linked Myotubular Myopathy
Wednesday, May 1, 2019 6:00 pm - 7:30 pm ET
Jefferson Room, Washington Hilton

The Audentes-sponsored symposium will provide an in-depth review of the latest developments in ASPIRO, the Phase 1/2 study of AT132 in patients with XLMTM.  Speakers will offer a range of clinical perspectives on the latest data and will include leaders from the fields of pediatric pulmonology, neurology, and muscle pathology.

Alan Boyd, MB, ChB, FRSB, FRCP, FFPM
Chief Executive Officer, Boyd Consultants, Crewe, UK
Fellow and Immediate Past President of the Faculty of Pharmaceutical Medicine at the Royal College of Physicians

Suyash Prasad, M.D.
Senior Vice President, Chief Medical Officer, Audentes Therapeutics

Mike Lawlor, M.D., Ph.D.
Associate Professor, Pediatric Pathology Clinical Neuromuscular Laboratory and Congenital Muscle Disease Tissue Repository, Medical College of Wisconsin

Geovanny Perez, M.D., MSc
Assistant Professor of Pediatrics & Pulmonary Medicine, George Washington University School of Medicine and Health Sciences 

Perry B. Shieh, M.D., Ph.D.
Associate Professor of Neurology, University of California Los Angeles

About X-linked Myotubular Myopathy 
X-linked Myotubular Myopathy (XLMTM) is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure, and early death. Mortality rates are estimated to be 50 percent in the first 18 months of life, and for those patients who survive past infancy, there is an estimated 25% mortality by the age of 10. XLMTM is caused by mutations in the MTM1 gene that lead to a lack or dysfunction of myotubularin, a protein that is needed for normal development, maturation, and function of skeletal muscle cells. The disease affects approximately 1 in 40,000 to 50,0000 newborn males.

XLMTM places a substantial burden of care on patients, families and the healthcare system, including high rates of healthcare utilization, hospitalization and surgical intervention. More than 80 percent of XLMTM patients require ventilator support, and the majority of patients require a gastrostomy tube for nutritional support. In most patients, normal developmental motor milestones are delayed or never achieved. Currently, only supportive treatment options, such as ventilator use or a feeding tube, are available.

About AT132 for the treatment of XLMTM 
Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of X-linked Myotubular Myopathy (XLMTM). AT132 may provide patients with significantly improved outcomes based on the ability of AAV8 to target skeletal muscle and increase myotubularin expression in targeted tissues following a single intravenous administration.

Audentes has reported promising safety, efficacy, and muscle biopsy data from ASPIRO, an ongoing, multicenter, ascending dose Phase 1/2 clinical study designed to evaluate the safety and efficacy of AT132. The preclinical development of AT132 was conducted in collaboration with Genethon (

AT132 has been granted Regenerative Medicine and Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA).

About Audentes Therapeutics, Inc. 
Audentes Therapeutics (Nasdaq: BOLD) is a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases.  We are leveraging our AAV gene therapy technology platform and proprietary manufacturing expertise to develop programs across three modalities: gene replacement, vectorized exon skipping, and vectorized RNA knockdown.  Our product candidates are showing promising therapeutic profiles in clinical and preclinical studies across a range of neuromuscular diseases. Audentes is a focused, experienced and passionate team driven by the goal of improving the lives of patients.

For more information regarding Audentes, please visit

Forward Looking Statements
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the potential benefits of AT132 and the expected timing for optimal dose selection and final agreement on regulatory submission pathways for AT132.  All statements other than statements of historical fact are statements that could be deemed forward-looking statements.  Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain.  Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercial its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission.  These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Audentes Contacts:

Investor Contact:
Andrew Chang

Media Contacts:
Sarah Spencer

Katie Hogan 

SOURCE Audentes Therapeutics, Inc.

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