Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
- Funder
- National Institutes of Health
- Award amount
- Not specified
- Deadline
- July 2, 2027
- Eligible countries
- United States
Summary
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.
Summary as listed on Grants.gov (opportunity 357570). Always read the full announcement for eligibility, limits and how to apply.
Read the full announcement on Grants.gov →What National Institutes of Health reviewers look for
- Importance of the research (significance and innovation), scored 1–9.
- Rigor and feasibility of the approach, scored 1–9. Preliminary data and alternative strategies carry much of this.
- Expertise and resources, rated sufficient or not, without a score.
- Overall impact: the likelihood the project will have a sustained, powerful influence on its field.
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