About X-Linked Myotubular Myopathy (XLMTM)
X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. It is caused by mutations in the�MTM1�gene, which encodes an enzyme called myotubularin. Myotubularin is thought to be involved in the development and maintenance of muscle cells. XLMTM affects approximately 1 in 50,000 newborn males worldwide.
AT001 is a novel drug candidate for the treatment of XLMTM based on adeno-associated virus (AAV) gene therapy technology. It comprises an AAV containing a full-length MTM1 cDNA (AAV-MTM1). Available data from animal studies suggest that gene therapy using AAV can lead to long-term gene expression, which may translate to a long-term treatment effect for patients. Audentes is developing AT001 in collaboration with Genethon.
About Audentes Therapeutics, Inc.
Audentes is a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases. The company consists of a focused, experienced, and passionate team driven by the goal of improving the lives of patients. Audentes takes pride in strong, global relationships with the patient, research, and medical communities.
For more information regarding Audentes, please visit�www.audentestx.com.
Genethon, located in Evry, France, is a non-profit R&D organization dedicated to the development of biotherapeutics for orphan genetic diseases, from research to clinical validation. Genethon specializes in the discovery and development of gene therapy drugs and has multiple ongoing programs at clinical, preclinical and research stages for neuromuscular, blood, immune system, liver, and eye diseases. To support clinical development of gene therapy drugs, Genethon has built one of the largest facilities worldwide for the production of clinical-grade gene therapy vectors.
For more information regarding Genethon, please visit www.genethon.fr.