At a glance
This NIH program funds preclinical proof-of-concept studies for rare diseases using established disease models to test whether a therapeutic candidate warrants further development. It also supports accompanying pharmacodynamic and pharmacokinetic studies, but not clinical trials or development of the model itself. Eligible applicants include U.S. higher education institutions, nonprofits, for-profits, governments, tribal organizations, faith-based or community-based organizations, regional organizations, and some federal entities; foreign organizations are not eligible, though foreign components are allowed and foreign subawards/subcontracts are not. The NIH expects to commit up to $1,200,000 in FY26, with 3 to 5 awards anticipated; the two-year project period may not exceed $275,000 in total direct costs, with no more than $200,000 in any single year. No cost sharing is required, and applications focused on rare cancers are not responsive.
What it funds
Official description from grants.gov
This notice of funding opportunity (NOFO) provides funding to conduct efficacy studies in an established rare disease preclinical model to demonstrate that a proposed therapeutic agent warrants further development. In addition to preclinical efficacy, accompanying pharmacodynamic and pharmacokinetic studies would be supported. Therapeutic agents include small molecules, biologics or biotechnology-derived products. The goal of this NOFO is to spur therapeutic development for a variety of rare diseases by advancing projects to the point where they would attract subsequent investment supporting full Investigational New Drug (IND) application development or progression to clinical trials in the case of repurposing or repositioning.
Who can apply
- City or township governments
- County governments
- For-profit organizations other than small businesses
- Independent school districts
- Native American tribal governments (Federally recognized)
- Native American tribal organizations (other than Federally recognized)
- Nonprofits with 501(c)(3) status (other than higher education)
- Nonprofits without 501(c)(3) status (other than higher education)
- Others
- Private institutions of higher education
- Public and State controlled institutions of higher education
- Public housing authorities / Indian housing authorities
- Small businesses
- Special district governments
- State governments