$75 Million

Audentes Therapeutics

At-the-market Offering

Audentes is a biotechnology company focused on developing and commercializing gene therapy products for patients suffering from serious, life-threatening rare diseases caused by a single gene defect. The Company has developed a proprietary gene therapy platform that aims to deliver a functional copy of a defected gene and the resulting production of the desired protein in target tissues. The company has developed four product candidates, AT132, AT342, and AT982 and AT307. Lead product candidate AT132 is in development for the treatment of X-Linked Myotubular Myopathy, or XLMTM. The FDA recently accepted the IND for AT132 and Audentes will initiate the Phase I/II ASPIRO study in 2017 and report topline data in 4Q 2017. The Company is simultaneously conducting two additional studies of AT132, a historical chart review and a Phase I/II run-in study with data expected from both studies in mid-2017.