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.


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 2020 Million Dollar Bike Ride.
Proposal Due Date: Monday, October 26, 2020 no later than 8pm (EST)
Full application documents and budget template are to be uploaded from our website, by invitation only.
FORMAT for documents:
Font and Page Margins: Use Arial typeface, a black font color, and a font size of 11 points. A symbol font may be used to insert Greek letters or special characters. Use 0.5 inch margins (top, bottom, left, and right) for all pages, including continuation pages. Print must be clear and legible; all text should be single-spaced.
Header: There should be a header at the top right on all pages of the PDF indicating the full name of the PI (e.g., PI: Smith, John D.).
For your convenience, a continuation page template is included at the end of the application document.
File names: ALL files to be uploaded should start with the LAST NAME of the PI followed by the brief name of the document. Examples: SMITH CV, SMITH Cover Page, SMITH Budget. If files are not labeled properly, you will be asked to resubmit the PDFs before your application can be considered.
CONTENT to be uploaded:
☐ Cover Page/Checklist/Institutional Signature Page [PDF].
☐ NIH-style Biosketch with Other Support of PI and key personnel (5 pages max). [PDF] The PI must include accurate and complete information regarding all other sources of grant support (current and pending), including title, abstract, annual and total amount of grant, inclusive funding period, and percent effort.
☐ Detailed Budget and Justification. [combined into one PDF] Complete Excel budget sheet (to be provided). Describe justifications in a Word document. Award will be for one year. Proposed funding period: February 1, 2021 – January 31, 2022.
 ☐ Research Plan (5 pages max) and Bibliography (1 page max). [combined into one PDF] Include the following sections: Specific Aims, Background and Significance, Preliminary Studies/Data, Research Design and Methods. Research plan should address the following questions: 1) Do you require access to reagents, cell lines, animal models, IRB/ethical board approvals, and/or equipment necessary to complete work? If so, please describe your plan to gain access within the time-frame of this grant period. 2) Have you identified qualified personnel to complete this project within the grant period? If not, please provide your plan to do so. Text citations should use a numbered format. Include all author names in the reference list. All previous MDBR grant awardees must include a statement of outcomes including publications, patents and additional funding granted as a result of data generated from those grants. Specific aims must be different from those in previous applications.
☐ Appendix [combined into one PDF] Limited to 5 pages of supplemental information pertaining to proposal or preliminary data only; a maximum of 3 relevant reprints are also acceptable. Include IRB and/or IACUC approval letters if relevant.

Orphan Disease Center