Gene therapy play Audentes raises $42.5M

Audentes Therapeutics Inc. (San Francisco, Calif.) raised $42.5 million in a series B round led by Deerfield Management. New investors Sofinnova Ventures and Venrock and existing investors OrbiMed, 5AM Ventures and Versant Ventures also participated. Audentes did not disclose whether the round is tranched.

Audentes is focused on developing gene therapies to treat rare musculoskeletal diseases. The company plans to start open-label Phase II trials of two compounds next year: AT001, which is an adeno-associated viral serotype 8 (AAV8) vector encoding the myotubularin 1 ( MTM1) gene to treat X-linked myotubular myopathy; and AT002, an AAV9 vector encoding the acid alpha glucosidase ( GAA) gene to treat Pompe's disease (see BioCentury, Oct 13). ...