Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
National Institutes of Health (NIH)
Total funding amount: up to $1,200,000
Project duration: maximum of 2 years
To conduct efficacy studies in established rare disease preclinical models to demonstrate that a proposed therapeutic agent warrants further development.
Researchers and organizations involved in rare disease therapeutics.
Patients affected by rare diseases.
Eligible organization types include higher education institutions, nonprofits, small businesses, local governments, state governments, and tribal governments.
Non-domestic (non-U.S.) entities are not eligible to apply.
Only U.S.-based organizations are eligible; foreign organizations and non-domestic components of U.S. organizations are not eligible.
Projects must focus on efficacy studies in established rare disease preclinical models.
Studies must demonstrate proof of concept for a proposed therapeutic agent.
The combined budget for direct costs may not exceed $275,000 over the two-year project period.
No more than $200,000 may be requested in any single year.
Application due date: June 2, 2026.
Opening date for submissions: May 2, 2025.
Applicants may submit more than one application, provided each is scientifically distinct.
Applications must include a Readiness of Agent attachment and a Partnership Plan attachment.
Applications will be evaluated based on significance, innovation, rigor, feasibility, and expertise.
Applications will undergo scientific peer review and will be assigned an overall impact score.
Scientific and technical merit, availability of funds, and relevance to program priorities will be considered.
Collaboration with a rare disease steering/oversight committee is required.
Funding will not be provided for model development.
Encouragement of multidisciplinary approaches in therapeutic development.
Applications that do not clearly address a rare disease will be considered non-responsive.
Demonstrating proof of concept in established rare disease models.
Submitting applications that do not comply with the application instructions.
Engage with potential licensing and commercialization partners early in the process.
Highlight the significance and innovation of the proposed therapeutic agent.