Synopsis

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.
Response Date	Jun 02, 2026 12:00:00 AM EDT
Posting Date	Dec 06, 2024 12:00:00 AM EST
Archive Date	Jul 08, 2026 12:00:00 AM EDT
Award Ceiling	N/a
Award Floor	N/a
Cost Sharing	No

Eligibility

Eligible Applicants

Public and State controlled institutions of higher education, Independent school districts, Public housing authorities/Indian housing authorities, State governments, Special district governments, County governments, Native American tribal organizations (other than Federally recognized tribal governments), Native American tribal governments (Federally recognized), Others (see text field entitled "Additional Information on Eligibility" for clarification), City or township governments, Small businesses, Private institutions of higher education, Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, For profit organizations other than small businesses, Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education

Additional Info on 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.

Additional Information

Agency Name	National Institutes of Health
Link to Additional Information:	https://grants.nih.gov/grants/guide/rfa-files/RFA-TR-25-002.html
Grantor Contact Information:	If you have difficulty accessing the full announcement electronically, please contact: GrantSolutions Help Desk Phone: 301-402-2541

CFDA Program(s)

• 93.350: National Center for Advancing Translational Sciences