The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania has announced a $10 million gift from an anonymous donor to create an interdisciplinary center focused on discovering novel treatments for orphan diseases.
The center will provide the core laboratories, techniques, collaborative relationships, and expertise for a coordinated international effort to eradicate so-called orphan diseases — those that affect fewer than two hundred thousand people — while fostering approaches to treatment that are common among various diseases. Working together with other academic institutions, the National Institutes of Health, the pharmaceutical industry, and philanthropic organizations, the center aims to reduce the technological and financial burdens of investigators working in isolation by supporting a range of collaborative approaches.
Among other things, its facilities will include 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 disease treatments. The center also will support collaborative approaches in biospecimen repositories for sample storage, genotyping and bioinformatics services, cell-based systems for developing new tests, nano-scale systems for developing new drug-delivery methods, and small and large animal models for testing potential 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, 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 nad private institutions — all designed to translate innovative research into the clinic. There's simply nothing else like it."