NCATS Preclinical Proof of Concept Studies for Rare Diseases
Funds U.S. researchers and companies conducting preclinical efficacy studies to advance therapeutic candidates for rare diseases toward IND applications.
NCATS Preclinical Proof of Concept Studies for Rare Diseases is an R21 exploratory grant administered by the National Center for Advancing Translational Sciences (NCATS) under the NIH. The program operates under FOA RFA-TR-25-002 (reissue of RFA-TR-24-023). Awards cover up to $275,000 in direct costs over a two-year project period, with no more than $200,000 in any single year. NCATS intends to co…
Rare diseases (affecting fewer than 200,000 people in the U.S.); small molecules, biologics, and biotechnology-derived products; preclinical efficacy, pharmacokinetics, and pharmacodynamics; drug repurposing and repositioning. Rare cancers are excluded (directed to NCI).
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