Orphan Disease Center Million Dollar Bike Ride Pilot Grant Program

The Orphan Disease Center Million Dollar Bike Ride Pilot Grant Program provides one-year research grants supporting rare diseases represented in the 2026 Million Dollar Bike Ride. Award availability and amounts are determined by fundraising totals associated with each participating disease team. For this funding round, the RFA identifies opportunities for CADASIL and Congenital Disorders of Glycosylation, or CDG. Although the Orphan Disease Center administers the grant program and review process, the respective rare disease team will execute the Notice of Award and is responsible for disbursing awarded funds. The CADASIL award is made possible by cureCADASIL and Million Dollar Bike Ride donors and participants, while the CDG award is made possible by Team CDG and Million Dollar Bike Ride donors and participants. The program supports research intended to advance understanding and treatment of the represented rare diseases. For CADASIL, cureCADASIL seeks work that advances understanding of disease mechanisms or clinical phenotyping that can facilitate future treatment trials, including biomarker or clinical predictor identification. Disease model initiatives and drug repurposing projects are also of interest, and both basic laboratory and clinical projects may be considered. For CDG, CDG CARE seeks basic, translational, and clinical research that advances understanding, diagnosis, clinical management, or treatment. Examples include disease mechanisms, biomarkers, natural history, model development, drug repurposing, precision medicine, novel therapeutics, gene-based therapies, and studies improving clinical care or clinical trial readiness. Projects should be structured so that the proposed work can be completed within the one-year award period. The RFA provides one CADASIL award of 106000 dollars and one CDG award of 50000 dollars, for a combined listed program total of 156000 dollars. Awards will not exceed the disease-specific totals. The proposed funding period is February 1, 2027 through January 31, 2028. Allowable direct costs include PI salary subject to the NIH Executive Level II salary cap, salary or stipend and related benefits for graduate students, postdoctoral fellows, or technical support, travel up to 1500 dollars, laboratory supplies and other research expenses, and consultant costs. Unallowable costs include tuition, professional membership dues, equipment costing more than 5000 dollars, general office supplies, institutional administrative charges, pre-award charges, indirect costs, and other expenses not directly related to the project. The RFA does not state a cost-share or matching requirement. Eligibility is global and international applicants are expressly invited to apply. Eligible applicants are individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a nonprofit institution or foundation. Prior Million Dollar Bike Ride award recipients may apply but must have current and updated project reporting to be eligible for selection. Applicants must also address their access to any necessary reagents, cell lines, animal models, ethical approvals, equipment, and qualified personnel needed to complete the work within the grant period. No-cost extensions are discretionary decisions of the disease team funding the project, are limited to one request per award, and may extend the project for no more than six months. Applicants must first submit a one-page Letter of Interest through the program website no later than September 18, 2026 at 8 PM EST. Full applications are by invitation only following review of the pre-application and are due October 19, 2026 at 8 PM EST. Full application files must be uploaded through the program website and must follow the required formatting and naming conventions, including Arial 11-point type, 0.5-inch margins, single spacing, a PI name header on every PDF page, and filenames beginning with the PI's last name. Required materials include the Cover Page, Checklist, and Institutional Signature Page; NIH-style biosketches with Other Support for the PI and key personnel; a detailed budget and justification; a research plan and bibliography; and an appendix when applicable. The research plan must include Specific Aims, Background and Significance, Preliminary Studies or Data, and Research Design and Methods, while also addressing access to required resources and availability of qualified personnel. Applications are reviewed for scientific content and relevance to the goals of the RFA through a two-step process. External reviewers first rate applications and assess strengths and weaknesses according to the stated review criteria. Funding recommendations are then developed from reviewer scores and written comments, with the final funding decision made by the applicable rare disease funding organization. Review criteria include scientific quality and budget justification, clarity of the study objective and hypothesis, scientific approach, clinical impact, research significance, investigator qualifications, and access to the resources and environment required to complete the project. Anonymous reviewer feedback may be shared upon applicant request at the discretion of the rare disease funding organization. The application and review process is confidential. The Orphan Disease Center states that Letter of Interest and full application content will remain confidential and that expert reviewers sign confidentiality agreements before review. Disease teams, as funding organizations, may access the application materials during review and are also bound by confidentiality requirements. Funds are issued through a cost reimbursement mechanism executed by the rare disease funding organization, with invoicing schedules and reporting requirements provided upon award. The stated program contact for additional information is Leslie Silverman at lsilve@upenn.edu. The RFA does not provide an explicit award notification date or state that this opportunity automatically recurs on an annual or other fixed schedule.

Propose a clearly defined project achievable within one year; align the project directly with the applicable disease research focus; clearly define the study objective and hypothesis; ensure the scientific approach addresses each specific aim; fully justify a reasonable budget; demonstrate investigator qualifications and access to required resources; do not request indirect costs; comply exactly with PDF formatting and PI-last-name file naming requirements