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Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)

National Institutes of Health · Opportunity number RFA-TR-25-002

View the official notice on Grants.gov
259 days left (deadline Jul 2, 2027)

Details summarized from the official notice as of Oct 15, 2026. Always confirm eligibility, deadlines and requirements in the official notice before applying.

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Key facts

Opportunity numberRFA-TR-25-002
AgencyNational Institutes of Health
Department of Health and Human Services
Assistance listing (ALN, formerly CFDA)93.350
PostedDec 6, 2024
DeadlineJul 2, 2027
Cost sharing or matching requiredNo
Funding typeGrant
CategoryHealth
Last updated hereOct 15, 2026

Summary from the notice

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

The official notice lists these applicant types as eligible:

  • Public and state colleges and universities
  • Public and Indian housing authorities
  • Nonprofits without 501(c)(3) status (other than colleges)
  • Federally recognized tribal governments
  • State governments
  • Special district governments
  • Nonprofits with 501(c)(3) status (other than colleges)
  • Other Native American tribal organizations
  • Private colleges and universities
  • Small businesses
  • For-profit organizations other than small businesses
  • County governments
  • Others named in the notice (see the eligibility text)
  • Independent school districts
  • City or township governments

Who should take a closer look

  • Small businesses
  • Nonprofits
  • Colleges and universities
  • State governments
  • Local governments
  • Tribal governments and organizations
  • Larger for-profit companies

Not listed as eligible

  • Individuals

Unless the eligibility text in the notice names you, this opportunity is probably not for you.

Eligibility details from the notice

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.

Registrations you will likely need

Most applications submitted through Grants.gov need the following. Registration can take several weeks, so start early. The notice lists the exact requirements.

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