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Penn launches center for treatment of orphan diseases, thanks to $10 million anonymous grant
PR Newswire
PHILADELPHIA — The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania has announced the launching of a first-of-its-kind interdisciplinary center focused on discovering novel treatments for orphan diseases. Formation of the new Penn Center for Orphan Disease Research and Therapy was catalyzed by a $10 million gift from an anonymous donor.
Diseases are classified as orphan when they affect fewer than 200,000 people. However, as there are approximately 7,000 diseases now identified in this population, more than 25 million Americans are currently afflicted. Many of these diseases are caused by genetic mutations. Research in many orphan diseases has lagged behind other major disease categories, such as diabetes and cardiovascular disease, in part because of a relative lack of technical expertise and funding mechanisms.
The Center will fill a crucial need by providing the core laboratories, techniques, collaborative relationships, and expertise to lead an international, coordinated effort in the eradication of orphan diseases. Key among these resources is a state-of-the-art, robotically controlled drug screening laboratory that enables researchers from around the world to rapidly probe existing compound libraries for effective, orphan disease treatments.
“The research and development marketplace is not designed to optimally support research to develop the therapies so desperately needed for orphan diseases,” said Glen N. Gaulton, PhD, executive vice dean and chief scientific officer at the Perelman School. “Penn’s new Center for Orphan Disease Research and Therapy will build not only strong collaborative relationships throughout Penn, but also with other leading academic medical centers, as well as public and private institutions — all designed to translate innovative research into the clinic. There’s simply nothing else like it.”
