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

National Institutes of Health · HHS

CFDA Numbers

93.350

Award Ceiling

—

Award Floor

—

Expected Awards

—

Close Date

Jun 2, 2026

Section I

How to Apply

Apply Online ↗

View on grants_gov ↗

Program Contact

NIH Grants Information<br/>grantsinfo@nih.gov
grantsinfo@nih.gov

Section II

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.

Eligible Applicant Types

00, 01, 02, 04, 05, 06, 07, 08, 11, 12, 13, 20, 22, 23, 25

Section III

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.

Section IV

Key Dates

Posted: Dec 6, 2024
Closes: Jun 2, 2026
Archive: Jul 8, 2026