Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
About
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.
The Preclinical Proof of Concept Studies for Rare Diseases grant program (R21 Clinical Trial Not Allowed) is designed for researchers and organizations seeking to advance therapeutic development for rare diseases. This funding opportunity supports efficacy studies conducted in established preclinical models to demonstrate that a proposed therapeutic agent warrants further development. Eligible therapeutic agents include small molecules, biologics, and biotechnology-derived products. Beyond preclinical efficacy testing, this grant also supports accompanying pharmacodynamic and pharmacokinetic studies. The primary goal of this program is to accelerate therapeutic development for rare diseases by advancing projects to a stage where they can attract subsequent investment for full Investigational New Drug (IND) application development or progression to clinical trials, particularly for repurposing or repositioning initiatives. This grant is ideal for researchers at academic institutions, eligible federal agencies, community-based organizations, and other qualified applicants who are working on innovative solutions to address unmet needs in rare disease treatment. If your organization is developing promising therapeutic candidates for rare diseases and needs funding to demonstrate preclinical proof of concept, this grant program offers a critical pathway to advance your research and increase competitiveness for future clinical development funding.
Funding Facts
● Applications openDeadline
July 2, 2027
Who can apply
Last verified July 5, 2026 · Source: Grants.gov
Grant Details
Funding Range
Varies by cycle
Deadline
2027-07-02
Grant Type
federal
Application Cycle
Annual
Best For
Researchers at academic institutions, HBCUs, minority-serving institutions, federal agencies, and community organizations developing therapeutic solutions for rare diseases
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 Govern
Frequently Asked Questions
Who is eligible for Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)?
Eligible applicants include: 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); and Indian/Native American Tribal Governments. For a complete and current list of eligible entities, applicants should consult the official funding opportunity announcement on the NIH website.
How much funding does Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed) provide?
Specific funding amounts for this grant cycle are not currently detailed in available information. Funding typically varies by cycle and project scope. Interested applicants should visit the official NIH Notice of Funding Opportunity (NOFO) page for current budget information and funding ranges.
How do I apply for Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)?
Applications for this NIH-sponsored grant program are submitted through the official NIH grants portal. Applicants should review the Notice of Funding Opportunity (NOFO) for detailed application instructions, required documentation, submission deadlines, and eligibility verification. Visit the NIH website directly to access the application system and complete guidance for this specific program.
What can Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed) funding be used for?
Funding supports efficacy studies in established rare disease preclinical models to demonstrate that a proposed therapeutic agent warrants further development. Allowable uses include preclinical efficacy testing, pharmacodynamic studies, and pharmacokinetic studies. Therapeutic agents may include small molecules, biologics, or biotechnology-derived products. The goal is to advance projects to the point where they attract investment for full IND application development or clinical trial progression. Clinical trials themselves are not permitted under this funding mechanism.
Last updated: July 5, 2026