Orphan Disease Center -- MPS I PILOT GRANT PROGRAM

Funding Agency:
University of Pennsylvania

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 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.

Letter of Interest are Due Tuesday, September 6, 2022 by 8 PM EST.

Agency Website

Eligibility Requirements

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.



Funding Type





Medical - Basic Science
Medical - Clinical Science
Medical - Translational

External Deadline

September 6, 2022