Mission
The Orphan Disease Center will develop transformative therapies using platform technologies that can be deployed across multiple rare diseases. We will emphasize disorders with substantial unmet need independent of their incidence and will strive to assure access to patients of all populations.
Impact
Each type of orphan disease affects such a small subset of the population, so the need for research and funding in this area is largely unmet. Our Center, the first of its kind, works closely with patient groups and foundations, pharma and biotech, and the academic community. We bring a unique set of programs to the table, enabling us to add value at any stage - from building the initial knowledge base to enabling therapeutic development. Through our grants, Programs of Excellence, International Patient Registries, Jump Start programs, and a number of new initiatives, the ODC seeks to drive therapeutic development for rare diseases. We help identify and fund the most promising therapeutics while also tackling obstacles present in rare disease drug development.
About Our Grantmaking
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 $17.2 million in rare disease research.
See Available Grants Below:
The ODC MDBR Pilot Grant Program provides a one‐year grant to support research related to a rare disease represented in the 2025 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team.
Eligibility
This RFA is open globally. International applicants are invited to apply. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. Prior MDBR award recipients must have current and updated project reporting to be eligible for selection.
This one-page LOI is due no later than Friday, September 19, 2025 by 8pm (EST).
Confidentiality:
The MDBR Grant Program is a confidential process and all content of the LOIs and Full Applications will be kept confidential by the ODC; our expert reviewers sign a CDA in advance of the review process. In order to encourage sharing of new techniques and findings to advance science, after funding decisions are made, the ODC will share a non-confidential lay summary of the research proposals received (required with your letter of interest), including those that were not funded, with each participating funding organization upon request.
Please review the RFA Guidelines before submitting your LOI.