As part of its therapeutic development projects for myotubular myopathy, and more recently, promising results from studies of the same treatment approach in a naturally‐occurring dog model of XLMTM (Childers et al., Science Translational Medicine, 22 January 2014 6:220ra10), Genethon started collaborating with Audentes Therapeutics, Inc. (biotechnology company dedicated to the development of innovative treatments for rare muscle diseases).
We have entered into an agreement to develop AT001 for the treatment of X‐Linked Myotubular Myopathy (XLMTM), a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment.
AT001 is a novel drug candidate based on adeno‐associated virus (AAV) gene therapy technology.
“Our agreement with Genethon is a significant step forward towards the development of a treatment for patients with this serious, rare disease,” said Matthew R. Patterson, President and CEO of Audentes. “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.”