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:

Overview: One-year grants up to $150,000 (direct costs) will be available to develop novel treatments for MPS I. The proposed therapeutic strategies should address the unmet needs of CNS manifestations of MPS I in patients with the severe and attenuated forms of the disease. Approaches that also address the somatic manifestations of the disease including the eye, bone, connective-tissue, and heart, which are not effectively treated with enzyme replacement therapy or bone marrow transplantation are welcome.

The applicant must have a proven track record in the development of novel therapeutics that progress toward clinical applications. The applicant must demonstrate utility of the proposed therapeutic platform in MPS I animal models during the course of the grant. A second year of funding is possible but should not be incorporated into the initial application.


All individuals holding a faculty‚Äźlevel appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA.

Letter of Intent (LOI) Form Due Date: Document is to be uploaded no later than 8pm (EST) on Tuesday, September 6, 2022.

Please review the RFA Guidelines before submitting your LOI.

To submit your LOI, please create a Submittable account OR log in to your existing account below.


Orphan Disease Center