Sarepta Therapeutics and University of Florida Partner to Accelerate the Discovery and Development of Therapies for Rare Genetic Diseases

CAMBRIDGE, Mass. and GAINESVILLE, Fla., Aug. 11, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and the University of Florida today announced a strategic collaboration to enable cutting-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at the University, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs.

“We have developed a productive incubator approach to our pipeline development, partnering with the best and brightest in genetic medicine, including leading academic researchers like those at the University of Florida, to discover and translate into meaningful therapies genetic medicine for rare diseases,” said Sarepta President and CEO Doug Ingram. “We are excited to partner with and support UF research that has the potential to profoundly improve and extend the lives of patients with rare genetic-based diseases.”

Through the collaboration, currently unique to UF, funding has been allocated for four innovative projects. These projects include exploratory research in novel gene therapy vectors, next-generation capsids and gene editing technologies as well as work in new therapeutic areas in degenerative genetic diseases. The goal is to foster early relationships with experts and accelerate the scientific advancements that lead to the development of transformational precision genetic medicines for patients in need.

“Our researchers intend to find solutions for diseases that have no cure or limited therapeutic options. Their goal is to move these solutions from their labs to patients who need them – to see their discoveries change lives. Because Sarepta has a focus and expertise in disease areas that coincide with the work of some of our scientists, it’s a match and collaboration that make sense and, we hope, will save lives,” said Jim O’Connell, assistant vice president of UF Innovate, the technology commercialization arm of the university. “Sarepta has a bold vision for transforming genetic disease because the company, ultimately, serves patients. That end goal drives its willingness and ability to translate research into a medical reality. We want to be part of that.”

University of Florida is a gene therapy powerhouse. UF researchers were the first to discover the life cycle of the adeno-associated virus (AAV), the smallest human virus. Using AAV as a benign delivery vehicle to carry therapeutics to a target, UF was first to reverse blindness in dogs with genetic disease, and UF researchers were integral in the first gene therapy approved by the FDA to treat an inherited genetic disease that can cause blindness. Today, UF is developing technologies in manufacturing, capsid design and therapies to address neuromuscular, cardiovascular, inflammatory, metabolic, pulmonary, skeletal, ophthalmic, and other disorders.

About Sarepta
At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on TwitterLinkedInInstagram and Facebook.

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