Promising preclinical data recently published in Science
“Our agreement with Genethon is a significant step forward towards the development of a treatment for patients with this serious, rare disease,” said
The development of a potential treatment for XLMTM using gene therapy technology was initiated at Genethon in 2009. Studies by Buj-Bello et al in a mouse model of the disease demonstrated 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 (Buj-Bello et al, Human Molecular Genetics, 2008, Vol. 17: 2132-2143; Buj-Bello et al, Molecular Therapy, 2013, 21:sup 1, S14). More recently, collaborators at Genethon, the
“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.”
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 1 in 50,000 newborn males worldwide. In a majority of newborns with the disorder, XLMTM causes death in the first years of life. There is currently no effective treatment for this disease.
Genethon, located in Evry,
For more information regarding Genethon, please visit www.genethon.fr.
About Audentes Therapeutics
Audentes™ is a private development stage biotechnology company committed to the development and commercialization of innovative new treatments for people with serious, rare muscle diseases through the application of adeno-associated virus (AAV) gene therapy technology. The company consists of a focused, experienced, and passionate team driven by the goal of improving 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.
GaËlle Monfort /