The University of Pennsylvania Orphan Disease Center’s 2017 Million Dollar Bike Ride Pilot Grant Program is now open! The Million Dollar Bike Ride (MDBR) Pilot Grant Program will provide a one-year grant to support research related to a rare disease represented in the 2017 Million Dollar Bike Ride. 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. Interested applicants must first submit a Letter of Interest (LOI), which is done by completing a brief online form, and uploading a 1‐page Pre‐Application. This LOI is due by Monday, September 18, 2017 by 8:00 p.m. EST. Full grant applications are accepted by invitation only after LOIs are approved.
The following lysosomal diseases were represented in the 2017 Million Dollar Bike Ride last May in Philadelphia, and their available grants are detailed:
Mucolipidosis Type IV (ML4): One $71,939 pilot grant available. This grant is offered to investigators conducting research on all aspects of ML4 disease, including disease pathogenesis and clinical studies. Preference will be given to those research projects developing new therapies for ML4, and translational research projects that improve our understanding of the disease state and pathogenesis, such as identifying biomarkers or functional outcome measures to assess therapeutic impact. This grant is made possible by the intrepid riders of Together4Ido, TeamCureML4, Climb4Carin, Pedal4Paul, Danny4theGirls, and MayaanHikes4Meira.
Mucopolysaccharidoses (MPS): Two $59,449 pilot grants are available. Applications directed to treating the central nervous system manifestations or enzyme replacement therapy antibody responses are sought. These two grants are made possible by Team MPS, the National MPS Society, and The Ryan Foundation.
Niemann Pick Type C (NPC): Two $49,645 pilot grants are available. Consideration will be given to research projects developing new therapies for NPC, as well as those designed to complement therapies presently in the pipeline. Consideration will also be given to gene therapy proposals; studies focused on problems, including psychiatric issues, impacting quality of life through the lifespan of the patient population; and projects that improve our understanding of the biology, pathogenesis and disease state and that have a direct impact on translation of new treatments to patients. These two grants are made possible by Team NPC, the Andrew Coppola Foundation, Jammin’ for JP, Chase the Cure and iPedal4Chad.
Tay-Sachs, Sandhoff, GM-1, or Canavan Disease: One $42,419 pilot grant is available focusing on forms of Tay-Sachs, Sandhoff, GM-1, or Canavan disease. Proposals are solicited for innovative research projects that involve basic research, translational studies or clinical studies relevant to these diseases. Projects may be focused on (1) pre-clinical and clinical research needs, such as clinical outcome measures, registries, animal models, or biomarkers; or (2) technology approaches such as stem cells, molecular chaperones, substrate inhibitors, small molecule drug screening, gene therapy, or novel drug delivery to the brain. This grant is made possible by Team NTSAD and the National Tay-Sachs & Allied Diseases Association.
Questions regarding the scientific content of potential research projects can be directed to Monique Molloy; administrative queries should be sent to Samantha Charleston at the University of Pennsylvania’s Orphan Disease Center.