NIH PAR · NCATS · R21
PAR-25-450: Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Reviewed by Dr. Meng ZhaoLast reviewed October 16, 2026Data refreshed October 16, 2026Editorial standards
This Notice of Funding Opportunity (NOFO) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare diseases research by enabling efficient and effective movement of candidate therapeutics or diagnostics toward clinical trials, and to increase their likelihood of success. This could be through the development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials.
- NOFO number
- PAR-25-450
- Type
- PAR
- Activity code
- R21
- Primary institute
- NCATS · National Center for Advancing Translational Sciences
- Clinical trials
- Not Allowed
- Posted
- Sep 22, 2025
- Opens
- Jan 17, 2026
- Expires
- Jul 21, 2028
PAR-25-450 due dates
4 application due dates remain before the NOFO expires. Dates marked * are NIH standard due dates. Check the related notices below for changes NIH made after posting.
| Application Due Date(s) | Renewal / Resubmission / Revision (as allowed) | AIDS - New/Renewal/Resubmission/Revision, as allowed | Scientific Merit Review | Advisory Council Review | Earliest Start Date |
|---|---|---|---|---|---|
| February 18, 2026 | March 17, 2026 | Not Applicable | July 2026 | October 2026 | December 2026 |
| June 17, 2026 | July 17, 2026 | Not Applicable | October 2026 | January 2027 | April 2027 |
| February 17, 2027 | March 17, 2027 | Not Applicable | July 2027 | October 2027 | December 2027 |
| June 17, 2027 | July 19, 2027 | Not Applicable | October 2027 | January 2028 | April 2028 |
| February 17, 2028 | March 17, 2028 | Not Applicable | July 2028 | October 2028 | December 2028 |
| June 20, 2028 | July 20, 2028 | Not Applicable | October 2028 | January 2029 | April 2029 |
What its predecessor PAR-23-159 funded
PAR-25-450 reissues PAR-23-159. Until awards appear under the new number, its funding history is the best guide to who wins under this program: 10 projects and $4.1M in FY2022–FY2026.
Projects funded
10
Distinct projects, FY2022–FY2026
New awards
10
Type 1 (new project) award records
NIH funding
$4.1M
Award dollars under PAR-23-159, FY2022–FY2026
Funding by fiscal year
| Fiscal year | Award records | New awards | Funding |
|---|---|---|---|
| FY2022 | 0 | 0 | $0 |
| FY2023 | 0 | 0 | $0 |
| FY2024 | 4 | 4 | $1.4M |
| FY2025 | 7 | 6 | $2.7M |
| FY2026 | 0 | 0 | $0 |
Institutions with the most funding
- Children's Hosp of Philadelphia$905K · 2 projects
- University of California, San Diego$850K · 2 projects
- University of California, San Francisco$462K · 1 project
- Duke University$456K · 1 project
- University of Colorado Denver$429K · 1 project
- University of Pennsylvania$402K · 1 project
- Myogene Bio, LLC$303K · 1 project
- Greenstone Biosciences, Inc.$303K · 1 project
Funding institutes
- NCATS$3.4M · 9
- NICHD$718K · 2
Activity codes funded
- R2111 records
Most recent new awards
- Patient-Centric Clinical Outcome Measures for Multiple Sulfatase Deficiency (MSD) (opens NIH RePORTER)
Children's Hosp of Philadelphia · 1R21TR006299-01A1 · NCATS · Notice Sep 3, 2025
$490KR21
- Natural History Study and Identification of Patient-Centric Clinical Outcome Assessments in TUBB4A-LD (NIH R21) (opens NIH RePORTER)
Children's Hosp of Philadelphia · 1R21HD120719-01 · NICHD · Notice Aug 29, 2025
$415KR21
- Assessment of restoration of an internally deleted dystrophin as a surrogate biomarker for future clinical trials of a gene editing therapy in Duchenne muscular dystrophy (opens NIH RePORTER)
Myogene Bio, LLC · 1R21HD117763-01 · NICHD · Notice Aug 20, 2025
$303KR21
- 4-hydroxymandelate for HPDL deficiency, a novel mitochondrial encephalopathy (opens NIH RePORTER)
University of California, San Diego · 1R21TR006111-01A1 · NCATS · Notice Aug 18, 2025
$399KR21
- Validating interferon score and phospho-STING level as disease biomarkers of COPA syndrome (opens NIH RePORTER)
University of California, San Francisco · 1R21TR005838-01 · NCATS · Notice Jul 17, 2025
$462KR21
- Trial readiness for LCAT deficiency (opens NIH RePORTER)
University of Pennsylvania · 1R21TR005433-01 · NCATS · Notice Jun 20, 2025
$402KR21
FY2026 closed on September 30, 2026, but NIH keeps posting that year's awards to RePORTER for several weeks. FY2026 figures reflect records available on October 16, 2026 and may rise slightly at the next refresh.
Participating institutes
2 NIH institutes and offices take applications under PAR-25-450. Contact the program officer at the institute that fits your research before applying.
- NCATS · National Center for Advancing Translational Sciences
- NICHD · Eunice Kennedy Shriver National Institute of Child Health and Human Development
Related notices
Aug 31, 2022 · NOT-OD-22-198
Implementation Changes for Genomic Data Sharing Plans Included with Applications Due on or after January 25, 2023. See Notice NOT-OD-22-198.
Aug 5, 2022 · NOT-OD-22-189
Implementation Details for the NIH Data Management and Sharing Policy. See Notice NOT-OD-22-189.
Award terms
- Application types allowed
- NewResubmission The OER Glossary and the How to Apply Application Guide provide details on these application types. Only those application types listed here are allowed for this NOFO.
- Award budget
- Applications can request budgets that are up to 275,000 direct costs for the 2-year project period of the award. No more than 200,000 direct costs may be requested in any single year.
- Project period
- The scope of the proposed project should determine the project period. The maximum project period is 2 years. NIH grants policies as described in the NIH Grants Policy Statement will apply to the applications submitted and awards made from this NOFO.
- Funds available and number of awards
- The number of awards is contingent upon NIH appropriations and the submission of a sufficient number of meritorious applications.
Summarized from the NOFO text; the full NOFO governs eligibility, budget, and review.
Related NOFOs
- Reissue of PAR-23-159.
Other open NOFOs for R21
- PAR-25-442Expanding the Target Landscape by Drugging the Undruggable (R21 Clinical Trial Not Allowed)NCATS · next due Feb 17, 2027
- RFA-TR-25-002Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)NCATS
- PA-25-304NIH Exploratory/Developmental Research Project Grant (Parent R21 Clinical Trial Not Allowed)NIH · next due Oct 16, 2026
- PAR-25-068Pilot Studies of Biological, Behavioral and Social Mechanisms Contributing to HIV Pathogenesis Within the Mission of NIDDK (R21 Clinical Trial Not Allowed)NIDDK · next due Oct 16, 2026
- PAR-25-357NEI Research Grant for Vision-Related Secondary Data Analysis (R21 Clinical Trial Not Allowed)NEI · next due Oct 16, 2026
- PA-25-306NIH Exploratory/Developmental Research Project Grant (Parent R21 Clinical Trial Required)NIH · next due Oct 16, 2026
About this page
GotGrant gathers the NOFO, its participation and change notices, and its NOSIs from the NIH Guide for Grants and Contracts, and counts funded projects from NIH RePORTER by the opportunity number each award was made under. Data refreshed October 16, 2026; the NIH Guide is the authoritative source. See our data methodology.