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American Society of Gene and Cell Therapy Annual Meeting, Washington

American Society of Gene and Cell Therapy Annual Meeting, Washington
April 25, 2019 SueCarr

Professor Alan Boyd will be chairing the Audentes Symposium session at the American Society of Gene and Cell Therapy (ASGCT) annual meeting in Washington on the 1st May 2019.

Audentes Therapeutics, Inc. is a leading AAV-based genetic medicines company focused on developing and commercialising innovative products for serious rare neuromuscular diseases, will be giving a number of 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 (1×1014 vg/kg), and up to 24-weeks in the earliest patients enrolled in dose Cohort 2 (3×1014 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, 20196:00 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 paediatric pulmonology, neurology, and muscle pathology.