Center for Orphan Disease Research and Therapy

Welcome to the Center for Orphan Disease Research and Therapy at the University of Pennsylvania

H. Lee Sweeney, Ph.D., William Maul Measey Professor of Physiology
Director, Center for Orphan Disease Research and Therapy

Orphan diseases represent a collection of disorders that afflict <200,000 individuals for any single disease type, and yet, as there are ~7,000 orphan diseases, in aggregate over 25 million US citizens suffer substantial morbidity and mortality. Despite this burden, research in most orphan diseases has lagged far behind other major areas due to a combination of technological and funding limitations.

The mission of the Penn Center for Orphan Disease Research and Therapy is to facilitate and expedite the development of novel therapies for orphan disorders/diseases.  The Center will achieve this mission through innovation of therapeutic strategies and translation of these into the clinic, building on partnerships among investigators, academic institutions, industry, and funding agencies.

The focus of the Center lies in partnership and leveraging of resources:

• Uniting investigators and clinicians within Penn and multiple institutions who are committed to treating and curing orphan disorders/diseases.
• Creating resources at Penn to enable discovery and preclinical development of potential therapies, as well as the clinical translation of those efforts.
• Extending approaches developed in one disorder to multiple others, and developing new technologically advanced research services to support this research.
• Providing a facile means for both small biotech and large pharmaceutical companies to partner with academic researchers in orphan disease research and therapeutic development.
• Linking academicians to both public and private foundations that support biomedical research for orphan diseases.

If you would like to receive News and Updates on the center, please email: CODRT@mail.med.upenn.edu .  Thank you.