Audentes: Muscular gene therapies

Audentes Therapeutics Inc. is developing gene therapies to treat rare musculoskeletal diseases, including one with no approved therapy and another where a gene therapy could provide increased efficacy, safety and convenience over a marketed enzyme replacement therapy.

ATX001 is focused on X-linked myotubular myopathy (XLMTM). ATX002 is focused on Pompe's disease. ...