The Orphan Disease Center MPS l Pilot Grant Program provides one-year grants of up to $150,000 (direct costs) to develop novel treatments for MPS l. 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.

Letters of interest (LOIs) are due no later than Tuesday, September 6, 2022 by 8 p.m. EST.

Grant criteria, the entire request for applications (RFA), and additional program details can be found here. 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. This RFA is open globally. International applicants are invited to apply.