RFA-TR-25-002-Full-Announcement.html

This is a Request for Applications (RFA) from the Department of Health and Human Services/National Institutes of Health seeking proposals for preclinical proof of concept studies focused on rare diseases. The funding opportunity (RFA-TR-25-002) supports efficacy studies using established rare disease preclinical models to evaluate therapeutic agents including small molecules, biologics, and biotechnology-derived products. The program falls under CFDA 93.350 - National Center for Advancing Translational Sciences (NCATS).

The opportunity is open to a wide range of applicants including higher education institutions, tribal governments, small businesses, nonprofits, and other organizations. The primary goal is to advance therapeutic development for rare diseases to the point where projects can attract further investment for full IND application development or progression to clinical trials for drug repurposing/repositioning. Studies must include both efficacy testing and accompanying pharmacodynamic/pharmacokinetic analyses. This is a R21 grant mechanism that does not allow clinical trials.