Million Dollar Bike Ride

2017 Million Dollar Bike Ride Pilot Grant Program

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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.

Research Funding Opps Seeking Applicants!

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The Perelman School of Medicine at the University of Pennsylvania’s Orphan Disease Center (“ODC”) sponsors a Million Dollar Bike Ride each May in Philadelphia. As the result of this year’s successful Million Dollar Bike Ride, the ODC is offering several research funding opportunities in the lysosomal diseases. These grants are open to the international community (they are not limited to University of Pennsylvania or the Children’s Hospital of Philadelphia). These lysosomal disease research funding opportunities are:

1. For mucolipidosis type IV (ML4), one $45,000 pilot grant available to investigators conducting research on all aspects of disease including disease pathogenesis and clinical studies. This grant is made possible by Team Cure ML4, Pedal4Paul, Climb4Carin, and the ML4 Foundation.

2. For the MPS diseases, two $52,000 pilot grants available. ODC is seeking applications directed to treating the central nervous system manifestations of these diseases. These grants are made possible by Team MPS, the National MPS Society and The Ryan Foundation.

3. For Niemann-Pick disease type C (NPC), two $51,000 pilot grants are available. ODC will give preference to research projects developing new therapies for NPC, and translational research projects that improve our understanding of the biology, pathogenesis and disease state (i.e., biomarkers or functional outcome measures to assess therapeutic impact). This grant is made possible by Team NPC, the Andrew Coppola Foundation, Jammin’ for JP, Chase the Cure and iPedal4Chad.

4. For Tay-Sachs, Sandhoff, GM-1 or Canavan disease, one $42,000 pilot grant is available focusing on forms of Tay-Sachs, Sandhoff, GM-1, or Canavan disease. ODC is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies relevant to the diseases mentioned above. 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, novel drug delivery to the brain. This grant is made possible by Team NTSAD and the National Tay-Sachs & Allied Diseases Association.

Eligibility for these funding opportunities is restricted to individuals holding a faculty-level appointment at an academic institution, or a senior scientific position at a non-profit institution or foundation.

Deadline to Apply: Submit your Letter of Interest (“LOI”) in the required online form no later than Friday, September 16, 2016 by 8 p.m. EST. Full application is by invitation only, after review of LOIs.

For the downloadable Million Dollar Bike Ride RFA Guidelines, the link to the online Letter of Interest submission form that must be used, the full list of all available Million Dollar Bike Ride funding opportunities (this includes many non-lysosomal diseases) and other information, visit: current Orphan Disease Center funding opportunities and read the entry entitled “2016 Million Dollar Bike Ride Grants.”

Questions regarding the scientific content of potential projects can be directed to Ms. Molloy at moniquek@nullexchange.upenn.edu; administrative queries should be sent to Ms. Charleston at scharle@nullupenn.edu.