Audentes Therapeutics, Inc., a biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, and Genethon, a non-profit organization dedicated to the research and development of biotherapies for orphan genetic diseases, announce that they 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.”
The development of a potential treatment for XLMTM using gene therapy technology was initiated at Genethon in 2009.
“We are proud of our initial research on this important potential treatment for XLMTM and are very encouraged by the recently published data,” explained Frédéric Revah, Chief Executive Officer of Genethon. “We are confident that this collaboration with the experienced team at Audentes will help us achieve our goal of bringing a treatment to patients as soon as possible.”