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

Rare Daily Staff

Sarepta Therapeutics said it has entered into a strategic collaboration with the University of Florida to enable cutting-edge research for novel medicines for rare genetic diseases.

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.

Sarepta is allocating funding to four projects under the collaboration: exploratory research in novel gene therapy vectors; next-generation capsids; gene editing technologies; and 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,” said Jim O’Connell, assistant vice president of UF Innovate, the technology commercialization arm of the university. “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.”

University of Florida 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 U.S. Food and Drug Administration to treat an inherited genetic disease that can cause blindness. Currently, UF is developing technologies in manufacturing, capsid design, and therapies to address neuromuscular, cardiovascular, inflammatory, metabolic, pulmonary, skeletal, ophthalmic, and other disorders.

Photo: Sarepta president and CEO Doug Ingram