NIH PAR · NINDS · U01
PAR-25-327: Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Reviewed by Dr. Meng ZhaoLast reviewed October 16, 2026Data refreshed October 16, 2026Editorial standards
The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare neurological or neuromuscular disorders. The goal of this announcement is to accelerate the development of a promising clinical candidate with robust biological rationale and demonstrated proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.
- NOFO number
- PAR-25-327
- Type
- PAR
- Activity code
- U01
- Primary institute
- NINDS · National Institute of Neurological Disorders and Stroke
- Clinical trials
- Optional
- Posted
- Dec 18, 2024
- Opens
- Jan 9, 2025
- Expires
- Oct 9, 2027
PAR-25-327 due dates
3 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 10, 2025 | February 10, 2025 | Not Applicable | July 2025 | October 2025 | December 2025 |
| June 09, 2025 | June 09, 2025 | Not Applicable | November 2025 | January 2026 | April 2026 |
| October 09, 2025 | October 09, 2025 | Not Applicable | March 2026 | May 2026 | July 2026 |
| February 09, 2026 | February 09, 2026 | Not Applicable | July 2026 | October 2026 | December 2026 |
| June 09, 2026 | June 09, 2026 | Not Applicable | November 2026 | January 2027 | April 2027 |
| October 09, 2026 | October 09, 2026 | Not Applicable | March 2027 | May 2027 | July 2027 |
| February 09, 2027 | February 09, 2027 | Not Applicable | July 2027 | October 2027 | December 2027 |
| June 09, 2027 | June 09, 2027 | Not Applicable | November 2027 | January 2028 | April 2028 |
| October 08, 2027 | October 08, 2027 | Not Applicable | March 2028 | May 2028 | July 2028 |
Projects funded under PAR-25-327
NIH RePORTER lists 4 projects funded under PAR-25-327 in FY2022–FY2026, with $5.4M in award dollars. Counts include each project's later non-competing years, which keep the original NOFO number.
Projects funded
4
Distinct projects, FY2022–FY2026
New awards
4
Type 1 (new project) award records
NIH funding
$5.4M
Award dollars under PAR-25-327, FY2022–FY2026
Funding by fiscal year
| Fiscal year | Award records | New awards | Funding |
|---|---|---|---|
| FY2022 | 0 | 0 | $0 |
| FY2023 | 0 | 0 | $0 |
| FY2024 | 0 | 0 | $0 |
| FY2025 | 0 | 0 | $0 |
| FY2026 | 4 | 4 | $5.4M |
Institutions with the most funding
- Massachusetts General Hospital$2.5M · 1 project
- University of Michigan at Ann Arbor$1.3M · 1 project
- Cedars-Sinai Medical Center$825K · 1 project
- University of California, San Francisco$650K · 1 project
Funding institutes
- NINDS$5.4M · 4
Activity codes funded
- U014 records
Most recent new awards
- Advancing Ex Vivo Gene Therapy: IND-Enabling Studies for ASAH1-related SMA-PME (opens NIH RePORTER)
Cedars-Sinai Medical Center · 1U01NS151207-01 · NINDS · Notice Sep 17, 2026
$825KU01
- IND-Enabling Studies of an Antisense Oligonucleotide Therapy for CLN3 Batten Disease (opens NIH RePORTER)
University of Michigan at Ann Arbor · 1U01NS148918-01 · NINDS · Notice Jul 14, 2026
$1.3MU01
- Targeted Gene Repair Therapy for Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS) Caused by ACTA2 R179H Mutation (opens NIH RePORTER)
Massachusetts General Hospital · 1U01NS142895-01A1 · NINDS · Notice May 5, 2026
$2.5MU01
- Prenatal AAV9 gene replacement therapy for patients with severe infantile GM1 gangliosidosis (opens NIH RePORTER)
University of California, San Francisco · 1U01NS146166-01 · NINDS · Notice Mar 23, 2026
$650KU01
What its predecessor PAR-22-030 funded
PAR-25-327 reissues PAR-22-030. Its funding history is the best guide to who wins under this program: 5 projects and $27.6M in FY2022–FY2026.
Projects funded
5
Distinct projects, FY2022–FY2026
New awards
5
Type 1 (new project) award records
NIH funding
$27.6M
Award dollars under PAR-22-030, FY2022–FY2026
Funding by fiscal year
| Fiscal year | Award records | New awards | Funding |
|---|---|---|---|
| FY2022 | 0 | 0 | $0 |
| FY2023 | 3 | 3 | $1.8M |
| FY2024 | 4 | 2 | $8.3M |
| FY2025 | 4 | 0 | $9.5M |
| FY2026 | 2 | 0 | $7.9M |
Institutions with the most funding
- Columbia University Health Sciences$17.5M · 2 projects
- University of Pennsylvania$6.0M · 1 project
- UT Southwestern Medical Center$2.1M · 1 project
- Broad Institute, Inc.$1.6M · 1 project
- Research Inst Nationwide Children's Hosp$376K · 1 project
Funding institutes
- NINDS$27.6M · 13
Activity codes funded
- U0113 records
Most recent new awards
- IND-enabling studies of non-immunogenic gene therapy for ultra-rare DMD patients excluded from dystrophin trials (opens NIH RePORTER)
University of Pennsylvania · 1U01NS134672-01A1 · NINDS · Notice Jul 30, 2024
$1.5MU01
- Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS (opens NIH RePORTER)
Columbia University Health Sciences · 1U01NS134684-01 · NINDS · Notice Feb 13, 2024
$5.2MU01
- Viral Gene Therapy for Menkes Disease (opens NIH RePORTER)
Research Inst Nationwide Children's Hosp · 1U01NS129343-01A1 · NINDS · Notice Aug 30, 2023
$376KU01
- IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial (opens NIH RePORTER)
UT Southwestern Medical Center · 1U01NS129339-01A1 · NINDS · Notice Aug 24, 2023
$632KU01
- Advancement of Prion Protein-Lowering Divalent siRNA Therapy for Prion Disease (opens NIH RePORTER)
Broad Institute, Inc. · 1U01NS132994-01 · NINDS · Notice Aug 1, 2023
$800KU01
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-25-327. Contact the program officer at the institute that fits your research before applying.
- NINDS · National Institute of Neurological Disorders and Stroke
- none
Related notices
Mar 31, 2025 · NOT-OD-24-084
This funding opportunity was updated to align with agency priorities. Carefully reread the full funding opportunity and make any needed adjustments to your application prior to submission.April 4, 2024 - Overview of Grant Application and Review Changes for Due Dates on or after January 25,…
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.
Award terms
- Application types allowed
- NewResubmissionRevision 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
- Application budgets are not limited but need to reflect the actual needs of the proposed project.
- Project period
- The scope of the proposed project should determine the project period. The project period must not exceed 3 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-22-030.
- Companion NOFOs: PAR-25-326.
Other open NOFOs for U01
- PAR-25-149Limited Competition: Renewal of Existing NINDS-Supported Clinical Trial Cooperative Agreement Awards (U01 - Clinical Trials Required)NINDS · next due Nov 5, 2026
- RFA-NS-26-001Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)NINDS · next due Nov 10, 2026
- RFA-NS-27-015BRAIN Initiative Connectivity across Scales (BRAIN CONNECTS): Specialized Projects for Scalable Technologies (U01 Clinical Trial Not Allowed)NINDS · next due Feb 8, 2027
- RFA-AI-27-014Development of Candidate Medical Countermeasures (MCMs) and Technologies Against Radiological/Nuclear or Chemical Threats (U01 Clinical Trial Not Allowed)NIAID · next due Nov 9, 2026
- PAR-26-116Opportunities for Collaborative Research at the NIH Clinical Center (U01 Clinical Trial Optional)OD · next due Feb 5, 2027
- RFA-MH-26-140BRAIN Initiative: Brain Behavior Quantification and Synchronization- Next Generation Sensor Technology Development (U01 Clinical Trial Optional)NIMH · next due Jun 15, 2027
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.