RFA-TR-23-016: Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
Closed
This grant is closed. A newer notice from the same program is open now: Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed) (RFA-TR-25-002). Status: Closes July 2, 2027 (237 days left). It is in our archive of National Institutes of Health notices from 2023.
National Institutes of Health offered grants of up to $275,000 to nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses. Applications closed on March 25, 2024.
From the official notice on Grants.gov, last changed March 25, 2024. Checked November 7, 2026.
- Award ceiling$275,000
- Cost sharingNot required
- Funding typeGrant
About this NIH notice
- Activity codeR21
- Clinical trialsNot allowed
- Funded byNCATS
- Median yearly award$223,699
- Typical project length2.5 years
- R21, Exploratory/Developmental Research Grant. Early, exploratory ideas that may not have preliminary data yet, usually for up to two years. Other open R21 notices.
Projects funded under RFA-TR-23-016 were run by National Center for Advancing Translational Sciences (NCATS, 4 projects). Contact the program officer at the institute that fits your project before you apply; the notice lists them.
Application due dates, page limits and budget limits are set in the notice. Read RFA-TR-23-016 in the NIH Guide.
Other notices with this title
| Notice | Posted | Deadline | Status |
|---|---|---|---|
| RFA-TR-25-002 | December 6, 2024 | Closes July 2, 2027 (237 days left) | Open |
| RFA-TR-24-023 | April 3, 2024 | Closed December 5, 2024 | Closed |
Key dates
| Posted | July 5, 2023 |
|---|---|
| Deadline | March 25, 2024 |
| Archived by Grants.gov | April 24, 2024 |
Who can apply
This opportunity was open to nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses.
All 15 applicant types the notice lists
- State governments
- County governments
- City or township governments
- Special district governments
- Independent school districts
- Public and State controlled institutions of higher education
- Native American tribal governments (Federally recognized)
- Public housing authorities/Indian housing authorities
- Native American tribal organizations (other than Federally recognized tribal governments)
- Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
- Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
- Private institutions of higher education
- For profit organizations other than small businesses
- Small businesses
- Others (see text field entitled "Additional Information on Eligibility" for clarification)
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 Institutions) 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.
Is this a realistic fit?
- Projects NIH funded under this notice had a median yearly award of $223,699; it allows up to $275,000 per award.
- Most of the money in the 2 programs behind this notice (73% over five years) went to colleges and universities.
- No cost sharing or match is required.
What the grant funds
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 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.
Funded under RFA-TR-23-016 so far
NIH funded 4 projects under RFA-TR-23-016 in fiscal 2024 to 2025, at 4 institutions. The median yearly award was $223,699.
| Project | Institution | Fiscal year | Award |
|---|---|---|---|
| Triacetin Treatment for Propionic Acidemia by Rebalancing the Acetyl-CoA/Propionyl-CoA Metabolism | Duke University | 2025 | $246,148 |
| Durability of a gene editing therapy that restores dystrophin in a humanized mouse model of Duchenne muscular dystrophy | Myogene Bio, LLC | 2025 | $201,250 |
| Effective treatment of cystinuria with a novel engineered cyst(e)ine-degrading human enzyme therapy | University of Texas at Austin | 2024 | $437,761 |
| Proof-of-concept for the use of enzyme replacement therapy as a lifesaving treatment for sphingosine phosphate lyase insufficiency syndrome | University of California, San Francisco | 2024 | $463,150 |
Who has won Mental Health Research Grants grants before
From fiscal 2021 to 2025, Mental Health Research Grants (93.242) awarded $8.8 billion across 6,785 grant awards, an average of $1.3 million per award.
| Recipient | State | Awards | Amount |
|---|---|---|---|
| Yale Univ | Connecticut | 185 | $371 million |
| University of Pittsburgh - of the Commonwealth System of Higher Education | Pennsylvania | 200 | $297 million |
| University of California, Los Angeles | California | 176 | $234 million |
| Icahn School of Medicine at Mount Sinai | New York | 144 | $212 million |
| The Washington University | Missouri | 149 | $211 million |
Who has won National Center for Advancing Translational Sciences grants before
From fiscal 2021 to 2025, National Center for Advancing Translational Sciences (93.350) awarded $3.1 billion across 867 grant awards, an average of $3.6 million per award.
| Recipient | State | Awards | Amount |
|---|---|---|---|
| University of Pittsburgh - of the Commonwealth System of Higher Education | Pennsylvania | 15 | $95.3 million |
| Vanderbilt University Medical Center | Tennessee | 10 | $94.6 million |
| Duke University | North Carolina | 11 | $88.9 million |
| University of North Carolina at Chapel Hill | North Carolina | 16 | $74.5 million |
| The Johns Hopkins University | Maryland | 20 | $70.7 million |
Applying now
This notice no longer takes applications. Apply under the newer notice, RFA-TR-25-002, which is open now.
The notice on Grants.gov keeps its full text and attachments, which is useful for planning a resubmission.
Questions people ask
Is RFA-TR-23-016 still open?
No. It closed on March 25, 2024. A newer notice is open: RFA-TR-25-002, closes July 2, 2027 (237 days left).
Who can apply for Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)?
National Institutes of Health accepted applications from nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses. The notice adds further conditions, so read its eligibility section.
What is the deadline for Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)?
Closed March 25, 2024. Applications are no longer accepted; the program's newer notices are on this page when there are any.
How much funding is available?
Awards of up to $275,000 each.
Who has been funded under RFA-TR-23-016?
NIH RePORTER lists 4 projects funded under RFA-TR-23-016 in fiscal 2024 to 2025, at 4 institutions. The most were at Duke University, Myogene Bio, LLC, University of Texas at Austin.
Who has won Mental Health Research Grants grants before?
The largest recipients over five fiscal years were Yale Univ, University of Pittsburgh - of the Commonwealth System of Higher Education, University of California, Los Angeles. The program made 6,785 awards worth $8.8 billion in that time.
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