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 News Release - December 13, 2017

Audentes Therapeutics Appoints Mark A. Goldberg, M.D. to its Board of Directors

SAN FRANCISCO, Dec. 13, 2017 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced the appointment of Mark A. Goldberg, M.D. to its board of directors.

"We are delighted to welcome Mark to our board of directors," stated Matthew R. Patterson, President and Chief Executive Officer of Audentes. "Mark is a recognized industry leader with a background in the development of novel therapies to treat severe rare diseases. His demonstrated track-record in clinical development, regulatory affairs and corporate strategy will be an important asset for Audentes as we continue to advance our pipeline of innovative gene therapy products."

Dr. Goldberg is a rare disease leader who brings over 20 years of experience developing and commercializing products to address serious unmet medical needs. Earlier in his career, Dr. Goldberg served as the Executive Vice President, Global Medical and Regulatory Strategy at Synageva, with responsibility for medical and regulatory affairs. He previously served as Senior Vice President for Clinical Development and Global Therapeutic Group Head for Oncology and Personalized Genetic Health at Genzyme Corporation, where he was responsible for leading clinical development teams for all drugs in development in those therapeutic areas. While at Genzyme he played a central role in the development and approval of multiple innovative rare disease therapeutics, including Fabrazyme®, Aldurazyme®, Myozyme® and Lumizyme®. Dr. Goldberg is a board-certified medical oncologist and hematologist, holds appointments at Brigham and Women's Hospital and the Dana Farber Cancer Institute, and received his A/B from Harvard College and his M.D. from Harvard Medical School.

"I am very pleased to join the Audentes board of directors during this transformational time for the company," commented Dr. Goldberg. "Audentes has firmly established itself as a leader in the emerging field of gene therapy and is well-positioned for meaningful upcoming catalysts, including preliminary efficacy data from its lead XLMTM program, the initiation of a Phase 1/2 study in its Crigler-Najjar program and the advancement of its Pompe disease and cardiac gene therapy programs. I look forward to working closely with the Audentes board and management team during what promises to be an exciting 2018."

About Audentes Therapeutics, Inc.

Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases. We are currently conducting a Phase 1/2 clinical study of our lead product candidate AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and have three additional product candidates in development, including AT342 for the treatment of Crigler-Najjar Syndrome, AT982 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

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 of preliminary data from ASPIRO, the Phase 1/2 study of AT132 to treat XLMTM, and the timing of initiating the Phase 1/2 clinical study of AT342. 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 commercial its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to 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.

Source: Audentes Therapeutics

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