PHILADELPHIA— The Gene Therapy Program Preclinical Vector Core in the Perelman School of Medicine at the University of Pennsylvania has been awarded a Gene Therapy Resource Program (GTRP) contract for a third consecutive five-year period from the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH).
The mission of the GTRP is to translate gene therapy research to the clinic. The $13 million contract will support the Core, which is directed by internationally renowned expert in gene therapy James M. Wilson, MD, PhD, the Rose H. Weiss Professor and Director of Penn’s Orphan Disease Center, and a professor of Medicine and Pediatrics, by providing preclinical vector production, analytics, and immunology services to approved scientific investigators from Penn and other institutions.
“The field of gene therapy is finally hitting its stride, and this is exactly the time for the NIH to be providing a robust infrastructure to promote the rapid acceleration of discoveries into clinical trials,” Wilson said. “The NHLBI is well positioned to do so through the GTRP, which we are delighted to participate in by providing high quality, state-of-the-art vectors for pre-clinical studies.”
The Core disseminates novel adeno-associated virus (AAV) vectors to researchers around the world. AAVs are small virus particles with a genome of single-stranded DNA that infect humans and some other primate species. AAVs are not currently known to cause disease. Recombinant AAV vectors are based on AAV viruses that have been engineered to express genes of interest including therapeutic genes for the treatment of inherited or acquired disease.
Over its first ten-year period of GTRP support, the Core completed over 100 requests for research-grade vector production and 10 requests for immunology services for developing gene therapies for a wide range of disorders, including cardiovascular, blood, lung, genetic, and infectious disease.
Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System and Penn’s Raymond and Ruth Perelman School of Medicine, founded in 1765 as the nation’s first medical school.
The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $550 million awarded in the 2022 fiscal year. Home to a proud history of “firsts” in medicine, Penn Medicine teams have pioneered discoveries and innovations that have shaped modern medicine, including recent breakthroughs such as CAR T cell therapy for cancer and the mRNA technology used in COVID-19 vaccines.
The University of Pennsylvania Health System’s patient care facilities stretch from the Susquehanna River in Pennsylvania to the New Jersey shore. These include the Hospital of the University of Pennsylvania, Penn Presbyterian Medical Center, Chester County Hospital, Lancaster General Health, Penn Medicine Princeton Health, and Pennsylvania Hospital—the nation’s first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.
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