SAN FRANCISCO, Nov. 29, 2016 /PRNewswire/ -- 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 it has successfully initiated large-scale cGMP (current Good Manufacturing Practice) production runs in its new, state-of-the-art manufacturing facility.
Matthew R. Patterson, President and Chief Executive Officer, stated, "The establishment of large-scale internal manufacturing is a key strategic milestone for Audentes. We began this effort in 2015 with the goal of retaining full control over costs, timelines and intellectual property associated with manufacturing our products. With the initiation of cGMP runs, we have translated this vision into a reality and taken a significant step toward achieving our mission of providing transformative therapies for patients."
The facility's initial cGMP production suite includes two 500-liter bioreactors for use with the company's proprietary serum-free suspension cell culture-based production system. This represents capacity to meet the material requirements for the clinical trials of the company's current product candidates. Planned future expansions will provide increased capacity to support both commercial manufacture and clinical supply for new development programs. In addition, Audentes has established robust in-house process and analytical development operations to ensure the long-term scalability and quality of its manufacturing operations.
The plant has been designed and commissioned as a multi-product facility intended to support commercial licensure by both the U.S. Food and Drug Administration and European Medicines Agency. The company intends to initiate clinical trials for two of its products, AT132 for X-Linked Myotubular Myopathy and AT342 for Crigler-Najjar Syndrome, in 2017 with material manufactured in this facility.
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 have four products in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), 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.
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 ability of Audentes to maintain control over its cost, timelines and intellectual property, the ability of Audentes to use material manufactured in its internal plant to treat patients in 2017, or at all, whether the FDA, EMA or similar regulatory bodies will license Audentes to manufacture product at its facility for clinical or commercial use, the ability of the company's in-house process and analytical development to ensure the long-term scalability and quality of its manufacturing operations, whether current manufacturing capacity is suffient to meet the needs of the Audentes development programs, or whether future expansion in the plant will be sufficient to meet the needs of new pipeline programs or commercial manufacturing. 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.