Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
NIH funds preclinical efficacy, pharmacodynamic, and pharmacokinetic proof-of-concept studies in established rare disease animal or other preclinical models for therapeutic agents to support later IND development or clinical translation.
⚑ R21; clinical trials not allowed · Rare disease preclinical proof-of-concept only; efficacy in an established preclinical model plus PK/PD · Foreign organizations are listed in the notice text but then stated as not eligible to apply; foreign components in U.S. applications are allowed · Award ceiling listed as $0
Unit fits — one characterization, each unit's own rules
| Physical Sciences & Engineering (demo) | 65 good | technical depth: minor; funds applied research |
| IPPRA | 45 partial | peripheral portfolio topic: public_health; social/behavioral work is none; funds applied research; biomedical core — IPPRA health lane is communication/crisis/policy (capped); capped at 45 (limited social-science role) |
| Tom Love Innovation Hub | 30 weak | funds applied research; deep-tech content |
Description
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.
Eligibility
Other Eligible Applicants include the following: Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Eligible Agencies of the Federal Government; Faith-based or Community-based Organizations; Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Indian/Native American Tribal Governments (Other than Federally Recognized); Non-domestic (non-U.S.) Entities (Foreign Organizations); Regional Organizations; Tribally Controlled Colleges and Universities (TCCUs) ; U.S. Territory or Possession; Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply.Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.Foreign components, as defined in the NIH Grants Policy Statement, are allowed.
Apply
View on Grants.gov → CONTACT: National Institutes of Health <grantsinfo@nih.gov>
Proposal brief SEE AN EXAMPLE →
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Proposal shell · National Institutes of Health conventions SEE AN NIH EXAMPLE →
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