The Orphan Disease Center (ODC) at the University of Pennsylvania is pleased to announce the Mucopolysaccharidosis Type I (MPS I) Pilot Grant Program. One-year grants up to $150,000 (direct costs) will be available to develop novel nucleic and cell based treatments for MPS I.

The proposed therapeutic strategies should address the 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 not effectively treated with enzyme replacement therapy are welcome. The following non-inclusive list of topics are responsive to this RFA:

  1. Genome and base editing strategies to correct the common non-sense mutations responsible for MPS I;
  2. Improvements with allogeneic or gene-modified autologous hematopoietic stem cell transplantation to increase efficacy and/or reduce toxicity;
  3. Nucleic acid delivery technologies with improved distribution throughout the CNS and acceptable toxicity profiles;
  4. Strategies to reduce toxicity of intrathecal AAV with demonstrated advantages over existing platforms/approaches using vectors expressing IDUA.

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.

This Request for Applications (RFA) is open to the international community.  You do not need to be based in the United States 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.  We ask that you please share this funding opportunity with your department or other relevant researchers. 

For more details about this grant program and how to apply, please visit the ODC website here.  Letters of Interest (LOIs) are due no later than Monday, September 10, 2018 by 8pm EST.  Please refer to our website for instructions on LOI submission.

Please contact Samantha Charleston at, or (215) 573-6822 with any questions.

Samantha Miller

Registry/Program Coordinator

Orphan Disease Center

University of Pennsylvania

Phone: 215-746-2704