Assuring access to transformative therapies for all rare disease communities.
Connecting the dots
Research and funding for orphan diseases is grossly unmet. ODC provides technological and educational resources, identifies funding opportunities, and fosters therapeutic development and innovative research.
Inaugural ODC Rare Disease Research Symposium
October 16 | Children's Hospital of Philadelphia (CHOP)
This one-day symposium brings together leading investigators, clinicians, translational scientists, and patient advocacy leaders from Penn, CHOP, and the broader rare disease community to discuss the latest advances in rare disease research and therapeutic development. The program is organized around key therapeutic modalities—Gene Therapy, Gene Editing, RNA Therapeutics, and Non-Genetic Approaches to Rare Diseases—with dedicated sessions on trial design and the role of the rare disease community in research. The day will close with a keynote by Dr. Kiran Musunuru on the future of rare disease medicine.
Join us for a day of scientific exchange, discussion, and networking aimed at accelerating collaborations and advancing therapeutic development for patients with rare diseases.
For more information and to register click here
Million Dollar Bike Ride Pilot Grant Program
The ODC is offering 2 research opportunities focusing on CADASIL & CDG. This program provides a one-year grant to support research related to a rare disease represented in the 2026 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team.
Letters of Interest due September 18, 2026
Open to the international research community
In the United States, orphan diseases represent a collection of disorders that afflict fewer than 200,000 individuals for any single disease type. There are more than 7,000 distinct orphan diseases.
In the aggregate, over 25 million people in the United States suffer substantial morbidity and mortality from orphan diseases. Despite this huge number, research in most disease types has lagged far behind other major areas due to a combination of technological and funding limitations.
The Orphan Disease Center of the University of Pennsylvania exists to facilitate and fund research, and develop transformative therapies for rare diseases with significant unmet need.
We aim to make rare disease research accessible, collaborative, and sustainable.
Programs & Initiatives
Grant Opportunities
The ODC offers over 50 grant opportunities in 30+ disease areas annually to researchers across the globe, as well as within the Penn and CHOP community. Since 2011, our grant programs have funded $37.6 million in rare disease research.
JumpStart
The JumpStart program serves to establish and progress research agendas in emerging and neglected rare diseases. The JumpStart program partners with patient groups and families to address gaps by identifying key opinion leaders and introducing new researchers to a disease.
Million Dollar Bike Ride
The ODC is proud to partner with 30+ disease teams made up of patients, caregivers, foundations and family members who tirelessly raise much-needed funds for research grants.
Diseases We Study
Amyotrophic Lateral Sclerosis
CDKL5 Deficiency Disorder (CDD)
Mucopolysaccharidoses
