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Gene Therapy Start-Up Audentes Therapeutics: FDA Orphan Drug Designation And $42.5 Million In Financing

December 4, 2014

Audentes Therapeutics is a San Francisco-based biotechnology company founded in November 2012. The company develops new treatments for rare muscle diseases through the use of Adeno-Associated Virus (AAV) gene therapy technology. Audentes” comes from the Latin word audeo, which means “to dare”, “to be brave or bold”.

Audentes Therapeutics has two lead rare disease programs in preclinical studies that are based on AAV gene therapy technology :

•   AT001 for X-linked Myotubular Myopathy (XLMTM)

•   AT002 for Pompe Disease.

BioSpace recently publishes an online list of the “Top 30 Life Science Start-ups To Watch in the U.S.” Audentes Therapeutics is ranked as #4. On December 2nd, Audentes Therapeutics raises $42.5 million in Series B financing. This funding will help the company to achieve clinical development milestones for its two lead rare disease programs.

XLMTM is a rare, inherited disorder that results in muscle weakness and respiratory impairment caused by mutations in the MTM1 gene, that encodes an enzyme call myotubalarin. Myotubularin is thought to be involved in the development and maintenance of muscle cells. XLMTM affects about 1/50,000 newborn males worldwide. The goal of gene therapy for XLMTM is “long-term expression of myotubularin after administration of a vector carrying the MTM1 gene. Increased levels of myotubularin are expected to produce improvement in symptoms related to the disease.” Audentes Therapeutics is developing AT001 for XLMTM in collaboration with Genethon, a French non-profit R&D organization specializing in discovering and developing gene therapies for orphan genetic diseases.

FDA Orphan Drug Product Database Record For Audentes Therapeutics

On December 3rd, the FDA grants Audentes Therapeutics Orphan Drug Designation (ODD) for its product candidate At001, a gene therapy product for XLMTM:

Generic Name: Non-replicating adeno-associated viral vector, serotype 8, expressing human myotubularin gene
Trade Name: At001
Date Designated: 12-03-2014
Orphan Designation: X-linked myotubular myopathy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Audentes Therapeutics, Inc. 101 Montgomery St., #2650 San Francisco, CA 94104



Audentes Therapeutics Pipeline

Myotubular Trust.

Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.


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