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: May 2, 2025 (Open date) | June 2, 2026 (Full application due) | June 2, 2027 (Full application due) | July 2, 2027 (Final application deadline)
Funding Amounts: Up to $275,000 in direct costs over 2 years; no more than $200,000 in any single year; 3–5 awards anticipated; total program funding up to $1,200,000 in FY26.
Summary: Supports preclinical efficacy, pharmacodynamic, and pharmacokinetic studies in rare disease models to advance therapeutics toward IND development or clinical trials.
Description
This NIH funding opportunity supports preclinical proof-of-concept studies for rare diseases (defined as affecting fewer than 200,000 individuals in the US) using validated in vivo or advanced in vitro disease models. The goal is to demonstrate that a proposed therapeutic agent (small molecule, biologic, or biotechnology-derived product) warrants further development, including progression to IND-enabling studies or clinical trials, especially for repurposing or repositioning. The program supports efficacy, pharmacodynamic (PD), and pharmacokinetic (PK) studies, but does not fund model development or clinical trials.
Projects should aim to generate robust data that will attract further investment or facilitate the transition to clinical development. Applications must include a plan for partnership with a rare disease steering/oversight committee and demonstrate the readiness of the therapeutic agent for efficacy testing.
