SAN FRANCISCO, --?Audentes Therapeutics, Inc.?(Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today provided an update following the receipt of minutes from its Regenerative Medicine and Advanced Therapy (RMAT)/Type B meeting with the U.S. Food and Drug Administration (FDA) held in December 2018, regarding the company's lead gene therapy candidate, AT132, for the treatment of X-linked Myotubular Myopathy (XLMTM). The goal of the meeting was to review nonclinical, clinical, and chemistry, manufacturing, and controls (CMC) data generated to date in the AT132 program, and to align with the FDA on program next-steps to enable submission of a Biologics License Application (BLA) for AT132. "We're pleased that our first discussion with the FDA under RMAT was both collaborative and productive, and provided a potential path toward BLA submission for AT132," stated Matthew R. Patterson, Chairman and Chief Executive Officer.? "We appreciate the constructive feedback provided by the Agency and look forward to working with the FDA to advance AT132 to registration as quickly as possible." Following the meeting, Audentes is proceeding with its previously announced plan to enroll an additional 3-5 patients in Cohort 2 (3x10¹⁴ vector genomes per kilogram dose level) of ASPIRO, the Phase 1/2 study of AT132.? Optimal dose selection is expected to occur in the second quarter of 2019, after the evaluation of the six-month biopsy results from the first three patients dosed in Cohort 2.? Subsequent to the determination of the optimal dose, Audentes plans to provide an updated data package to FDA to facilitate final agreement on the path to BLA submission.? Audentes also received preliminary feedback on its CMC characterization and validation plans and will submit the requested information to the Investigational New Drug (IND) application on a continuing basis. In pursuit of global regulatory approvals for AT132, Audentes also initiated discussions with the European Medicines Agency (EMA) in the fourth quarter of 2018 under the recently granted Priority Medicines (PRIME) designation.? In the first quarter of 2019, Audentes anticipates receiving scientific advice from the EMA that will begin to define the path toward submission of a European Marketing Authorization Application. About AT132 for X-linked Myotubular Myopathy AT132 is the Audentes product candidate being developed to treat XLMTM, a disease caused by mutations in the MTM1 gene, which encodes the protein myotubularin.? Myotubularin plays an important role in the development, maintenance and function of skeletal muscle cells.? AT132 is comprised of an AAV8 vector containing a functional copy of the MTM1 gene.? Over the course of 2018, Audentes reported promising safety, efficacy and muscle biopsy data from ASPIRO, a multicenter, ascending dose Phase?1/2?clinical study designed to evaluate the safety and efficacy of AT132 in approximately 12 XLMTM patients less than five years of age.? The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr). AT132 has been granted RMAT, Rare Pediatric Disease, Fast Track and Orphan Drug designations by the?FDA, and PRIME and Orphan Drug designations by the EMA. About Audentes Therapeutics, Inc. Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases.? We are currently conducting Phase 1/2 clinical studies of our lead product candidates, AT132 for the treatment of XLMTM, and AT342 for the treatment of Crigler-Najjar syndrome.? We have two additional product candidates in development, AT845 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT).? We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities. For more information regarding Audentes, please visit www.audentestx.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the timing and nature of clinical development activities; the timing of regulatory interactions, submissions or approvals; and, the expected safety and efficacy profile of the company's product candidates.? All statements other than statements of historical fact are statements that could be deemed forward-looking statements.?Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercialize its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, establish and scale-up manufacturing processes that comply with regulatory requirements, protect intellectual property and other?risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the?Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Audentes Contacts: Investor Contact: Andrew Chang 415.818.1033 achang@audentestx.com Media Contact: Katie Hogan 415.951.3398 khogan@audentestx.com  

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