Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
Funding available for preclinical studies in rare diseases to show effectiveness of proposed treatments for potential further development into clinical trials.
Funder: National Institutes of Health
Due Dates: June 2, 2025 (New, Renewal, Resubmission) | May 1, 2025 (Letter of Intent) | June 2, 2026 (New, Renewal, Resubmission) | May 1, 2026 (Letter of Intent)
Funding Amounts: Up to $275,000 direct costs over 2 years (max $200,000 in any single year); 3–5 awards anticipated; total NIH commitment up to $1.2M in FY26.
Summary: Supports preclinical efficacy, pharmacodynamic, and pharmacokinetic studies of therapeutic agents in established rare disease models to advance candidates toward IND-enabling development or clinical trials.
Key Information: Clinical trials are not allowed; model development is not supported; a partnership plan with a rare disease steering/oversight committee is required.
Description
This NIH opportunity funds preclinical proof-of-concept studies for rare diseases, aiming to bridge the gap between early discovery and clinical development. The program supports efficacy studies in established and validated rare disease models (in vivo or advanced in vitro), as well as accompanying pharmacodynamic (PD) and pharmacokinetic (PK) studies. The goal is to generate robust data that will attract further investment for full IND-enabling studies or clinical trials, including for repurposed or repositioned therapeutics.
Therapeutic agents may include small molecules, biologics, or biotechnology-derived products. The program is administered by the National Center for Advancing Translational Sciences (NCATS).
Note: This opportunity does not support clinical trials or the development of new disease models. Applications focused on rare cancers are not eligible.
