SAN FRANCISCO, CA – September 24, 2014 – Audentes Therapeutics, Inc., a private biotechnology company dedicated to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced that Matthew Patterson, President and Chief Executive Officer, will present a corporate overview at the Leerink Partners Rare Disease Roundtable in New York City on October 1, 2014.
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 has two products in development, AT001 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and AT002 for the treatment of Pompe Disease. 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.
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. Audentes is developing AT001 for the treatment of XLMTM in collaboration with Genethon (www.genethon.fr). AT001 is a novel product based on AAV gene therapy technology.
About Pompe Disease
Pompe Disease is a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It is caused by mutations in a gene that encodes an enzyme called acid alpha-glucosidase (GAA), which is needed by the body to break down glycogen – a stored form of sugar used for energy. Pompe Disease affects approximately 1 in every 40,000 births. AT002 is a novel product based on AAV gene therapy technology.