Audentes Therapeutics Joins Global Organizations In Recognizing Rare Disease Day® 2018

February 28, 2018 8:00 AM

SAN FRANCISCO, Feb. 28, 2018 /PRNewswire/ -- 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 announced its support for Rare Disease Day®. The global theme of this year's Rare Disease Day is research. Rare disease research contributes to the development of diagnostic tools, treatments and cures, as well as improved health and social care for patients and their families.

"Audentes is proud to join the global rare disease community to recognize and support Rare Disease Day 2018," said Matthew R. Patterson, President and Chief Executive Officer of Audentes. "By raising awareness, we improve the chances that new treatments are made available for the millions of people who live with these diseases every day."

Audentes will be supporting Rare Disease Day through employee activities that remind us about everyday life for patients and their families touched by a rare disease. Audentes employees embrace the company's mission of bringing innovative gene therapy products to patients living with serious, life-threatening rare diseases and the day's activities will serve as a reminder that our work is vital and urgent.

About Rare Disease Day Rare Disease Day takes place on the last day of February, a month known for having a 'rare' number of days. It was first launched in Europe by EURORDIS and its Council of National Alliances in 2008 and is now observed in more than 94 countries. Rare Disease Day is sponsored in the United States by the National Organization for Rare Disorders (NORD)®, a leading independent, non-profit organization committed to the identification, treatment and cure of rare diseases.

For more information about Rare Disease Day in the United States, go to For information about global activities, go to

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 X-Linked Myotubular Myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar Syndrome. We have two additional product candidates in development, including 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

About AT342 for X-Linked Myotubular MyopathyAT132 is the Audentes product candidate being developed to treat XLMTM, a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age. XLMTM is 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. In January 2018, Audentes reported positive interim data from the first dose cohort of ASPIRO, a multicenter, ascending dose Phase 1/2 clinical study to evaluate the safety and preliminary 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 (

About AT342 for Crigler-Najjar Syndrome AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene for the treatment of Crigler-Najjar Syndrome, a rare monogenic disease characterized by severely high levels of unconjugated bilirubin in the blood and risk of irreversible neurological damage and death. The current standard of care for Crigler-Najjar Syndrome is daily, persistent phototherapy, usually for longer than 10 to 12 hours per day. Phototherapy wanes in effectiveness as children age, and a liver transplant may be required for survival. Data from LUSTRO, a prospective natural-history run-in study in Crigler-Najjar patients, demonstrate that even with strict adherence to a persistent daily phototherapy regimen, bilirubin may only be reduced to levels just below those considered to be neurotoxic. In February 2018 Audentes announced it had initiated VALENS, a multicenter, ascending dose Phase 1/2 clinical study to evaluate the safety and preliminary efficacy of AT342 in approximately 12 Crigler-Najjar patients greater than or equal to one year of age.

Forward Looking StatementsThis 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 key program milestones, including plans to dose patients in, and report clinical data from, ASPIRO, the Phase 1/2 study of AT132 in XLMTM, and VALENS, the Phase 1/2 study of AT342 in Crigler-Najjar Syndrome. 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.

Audentes Contacts:Investor Contact:Andrew Chang415.818.1033

Media Contact:Paul Laland415.519.6610

Audentes Therapeutics, Inc.

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