Audentes Therapeutics Inks $30M in Series A

Audent Therapeutics, Inc. A recently founded biotechnological company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of the $30 million Series A funding The funding was led by OrbiMed Advisors with the participation of 5AM Ventures and Versant Ventures. This funding will allow the Company to further advance its two leading programs, AT001 for X-linked Myotubular Myopathy (XLMTM) and AT002 for Pompe Disease, and to evaluate additional candidate programs. Both AT001 and AT002 are innovative, novel treatments based on adeno-associated virus (AAV) technology for gene therapy.

‘This funding represents a major step forward in developing new potential treatments for patients affected by very serious orphan muscle diseases. I’m pleased to have the opportunity to work with such a fantastic group of people who share our vision for success in bringing these therapies to patients,” said Matthew R. Patterson, President and Chief Executive Officer of Audents ‘Our dedicated, experienced team is committed to advancing a promising scientific approach to gene therapy and looks forward to working closely with our patients, research and medical community partners in this process. ”

Audentes, based in San Francisco, CA, was founded by Mr. Patterson, in collaboration with Thomas J, in November 2012. Schutz, M D. D. Ph, Ph. D. D. And the OrbiMed Advisers The Board of Directors of Audentes will be composed of Jonathan Silverstein (OrbiMed Advisors), who will serve as Chairman, Kush Parmar, M. D. D. Ph, Ph. D. D. 5AM Ventures, Thomas Woiwode, Ph.D. D. D. Dr. (Versant Ventures) Schuetz, who is going to act as an independent director, and Mr. Patterson’s

X-Linked Myotubular Myopathy (XLMTM)

X-Linked Myotubular Myopathy (XLMTM) is a rare hereditary disorder characterized by severe muscle weakness and respiratory impairment. It is caused by mutations in the MTM1 gene that encode an enzyme called myotubularin. Myotubularin is believed to be involved in the development and maintenance of muscle cells. XLMTM affects about 1 in 50,000 newborn males worldwide.

About Pump Disease

Pompe disease is a rare, inherited disorder characterized by progressive muscle weakness and impaired respiratory function. It is caused by mutations in a gene that encodes an enzyme called alpha-glucosidase acid (GAA) that the body needs to break down glycogen-a stored form of sugar used for energy purposes. Pompe disease affects about 1 in every 40,000 births.

About Audents Therapeutics, Inc.

AudentesTM is a biotechnology company committed to developing and commercializing innovative new treatments for people with severe, rare muscular diseases through the use of Adeno-associated Virus (AAV) gene therapy technology. The company consists of a dedicated, experienced and passionate team driven by the goal of improving the lives of patients. Audentes is proud of its strong, global relationship with patients, research and the medical community.

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