NCATS Preclinical Proof of Concept Studies for Rare Diseases (R21)
NCATS, NIH (RFA-TR-25-002)
Funds efficacy studies of a proposed therapeutic in an established rare-disease model, with PK/PD work allowed, to justify further development toward an IND. Roughly 3–5 awards are made per year.
TAFAZZIN gene therapy, mitochondria-targeted agents, and cardiolipin-targeted candidates tested in tafazzin-knockout models are within scope.
US higher-education institutions, nonprofits, for-profits, and governments. US organizations only — foreign organizations and foreign subawards are ineligible, though foreign components (as NIH defines them) are allowed.
Via eRA Commons / Grants.gov / ASSIST under RFA-TR-25-002; the deadline was extended to Jun 2, 2027.
/grants/ncats-preclinical-proof-of-concept-studies-for-rare-diseases-r21/
Last updated August 28, 2026 · Suggest an update to this entry