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news.US-based biotechnology firm Audentes Therapeutics and Genethon, a non-profit organization dedicated to the research and development of biotherapies for orphan genetic diseases, have entered into an agreement to develop AT001 for the treatment of X-Linked Myotubular Myopathy (XLMTM).
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XLMTM is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment.
The company said that AT001 is a novel drug candidate based on adeno-associated virus (AAV) gene therapy technology.
Audentes president and CEO Matthew Patterson said the agreement with Genethon is a significant step forward towards the development of a treatment for patients with this serious, rare disease.
"The combination of Genethon’s expertise in the manufacturing and development of gene therapy products and Audentes’ world-class orphan drug development team will allow us to rapidly advance this program," Patterson said.
Development of a possible treatment for XLMTM using gene therapy technology was started at Genethon in 2009.
The studies carried out by Buj-Bello et al in a mouse model of the disease showed that delivery of the deficient gene using an AAV vector system resulted in increased expression of the protein, improvement in muscle architecture, reversal of muscle hypotrophy, improvement in muscle strength, and an improvement in overall survival.
The company said that recently, collaborators at Genethon, the University of Washington and Harvard University released positive results from studies of the same treatment approach in a naturally-occurring dog model of XLMTM.
The studies showed 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.
The data from the studies 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.