The National Institutes of Health is offering this grant to support preclinical proof-of-concept studies for rare diseases. This funding specifically targets efficacy studies in established rare disease models, demonstrating that proposed therapeutic agents (small molecules, biologics, or biotechnology products) warrant further development. Accompanying pharmacodynamic and pharmacokinetic studies are also supported. The grant’s primary purpose is to accelerate therapeutic development for various rare conditions. By advancing projects to a mature stage, it seeks to attract subsequent investment vital for Investigational New Drug (IND) application development or progression to clinical trials, especially for repurposed or repositioned agents.
Opportunity ID: 357570
General Information
| Document Type: | Grants Notice |
| Funding Opportunity Number: | RFA-TR-25-002 |
| Funding Opportunity Title: | Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed) |
| Opportunity Category: | Discretionary |
| Opportunity Category Explanation: | – |
| Funding Instrument Type: | Grant |
| Category of Funding Activity: | Health |
| Category Explanation: | – |
| Expected Number of Awards: | – |
| Assistance Listings: | 93.350 — National Center for Advancing Translational Sciences |
| Cost Sharing or Matching Requirement: | No |
| Version: | Synopsis 1 |
| Posted Date: | Dec 06, 2024 |
| Last Updated Date: | Dec 06, 2024 |
| Original Closing Date for Applications: | Jun 02, 2026 |
| Current Closing Date for Applications: | Jun 02, 2026 |
| Archive Date: | Jul 08, 2026 |
| Estimated Total Program Funding: | – |
| Award Ceiling: | – |
| Award Floor: | – |
Eligibility
| Eligible Applicants: | Independent school districts Native American tribal organizations (other than Federally recognized tribal governments) Public and State controlled institutions of higher education For profit organizations other than small businesses Special district governments Small businesses County governments Private institutions of higher education City or township governments State governments Others (see text field entitled “Additional Information on Eligibility” for clarification) Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education Native American tribal governments (Federally recognized) Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Public housing authorities/Indian housing authorities |
| Additional Information 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 |
| 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. |
| 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:
NIH Grants Information
grantsinfo@nih.gov Email:grantsinfo@nih.gov |
Version History
| Version | Modification Description | Updated Date |
|---|---|---|
Related Documents
Folder 357570 Full Announcement-RFA-TR-25-002 -> RFA-TR-25-002-Full-Announcement.pdf
Packages
| Agency Contact Information: | NIH Grants Information grantsinfo@nih.gov Email: grantsinfo@nih.gov |
| Who Can Apply: | Organization Applicants |
| Assistance Listing Number | Competition ID | Competition Title | Opportunity Package ID | Opening Date | Closing Date | Actions |
|---|---|---|---|---|---|---|
| FORMS-I | Use for due dates on or after January 25, 2025 | PKG00289494 | May 02, 2025 | Jun 02, 2026 | View |
Package 1
Mandatory forms
357570 RR_SF424_5_0-5.0.pdf
357570 PHS398_CoverPageSupplement_5_0-5.0.pdf
357570 RR_OtherProjectInfo_1_4-1.4.pdf
357570 PerformanceSite_4_0-4.0.pdf
357570 RR_KeyPersonExpanded_4_0-4.0.pdf
357570 PHS398_ResearchPlan_5_0-5.0.pdf
357570 PHSHumanSubjectsAndClinicalTrialsInfo_3_0-3.0.pdf
Optional forms
357570 RR_Budget_3_0-3.0.pdf
357570 RR_SubawardBudget30_3_0-3.0.pdf
357570 PHS398_ModularBudget_1_2-1.2.pdf
357570 PHS_AssignmentRequestForm_4_0-4.0.pdf
Share This Post, Choose Your Platform!
About the Author: osnqt
