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

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

Agency: National Institutes of Health

Assistance Listings: 93.350 -- National Center for Advancing Translational Sciences

Last Updated: June 5, 2026

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

Eligible applicants

Government

City or township governments
Special district governments
Federally recognized Native American tribal governments
County governments
Public and Indian housing authorities
State governments

Business

For-profit organizations other than small businesses
Small businesses

Education

Independent school districts
Private institutions of higher education
Public and state institutions of higher education

Miscellaneous

Other

Nonprofit

Nonprofits non-higher education without 501(c)(3)
Other Native American tribal organizations
Nonprofits non-higher education with 501(c)(3)

Additional information

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.

Grantor contact information

Description

NIH Grants Information grantsinfo@nih.gov

Email

See Section VII. Agency Contacts within the full opportunity announcement for all other inquires.

grantsinfo@nih.gov

Documents

File name	Description	Last updated
RFA-TR-25-002-Full-Announcement.html	RFA-TR-25-002 Full Announcement Text	Dec 6, 2024 05:05 PM UTC

Link to additional information

https://grants.nih.gov/grants/guide/rfa-files/RFA-TR-25-002.html

Closing: July 2, 2027

Application process

This site is a work in progress. Go to www.grants.gov to apply, track application status, and subscribe to updates.

Award

$--

Program Funding

Expected awards

$--

Award Minimum

$--

Award Maximum

Funding opportunity number:

RFA-TR-25-002

Cost sharing or matching requirement:

Funding instrument type:

Grant

Opportunity Category:

Discretionary

Opportunity Category Explanation:

Category of Funding Activity:

Health

Category Explanation:

History

Version:

Posted date:

December 6, 2024

Archive date:

August 1, 2027