PAR-22-030: Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Closed
This grant is closed. A newer notice from the same program is open now: Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) (PAR-25-327). Status: Due dates in the notice, expires October 8, 2027. It is in our archive of National Institutes of Health notices from 2021.
National Institutes of Health offered grants to nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses. Applications closed on October 11, 2024.
From the official notice on Grants.gov, last changed December 1, 2023. Checked November 7, 2026.
- Award amountsNot given on Grants.gov
- Cost sharingNot required
- Funding typeCooperative Agreement
About this NIH notice
- Activity codeU01
- Clinical trialsOptional
- Funded byNINDS
- Median yearly award$1.5 million
- Typical project length3 years
- U01, Research Project Cooperative Agreement. Like an R01, but NIH staff are substantially involved in the work. Other open U01 notices.
Projects funded under PAR-22-030 were run by National Institute of Neurological Disorders and Stroke (NINDS, 5 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 PAR-22-030 in the NIH Guide.
Other notices with this title
| Notice | Posted | Deadline | Status |
|---|---|---|---|
| PAR-25-327 | December 18, 2024 | Due dates in the notice, expires October 8, 2027 | Open |
Key dates
| Posted | November 9, 2021 |
|---|---|
| Deadline | October 11, 2024 |
| Archived by Grants.gov | November 10, 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?
- Most of the program's money (76% over five years) went to colleges and universities.
- No cost sharing or match is required.
What the grant funds
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 funding opportunity announcement (FOA) 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.
Funded under PAR-22-030 so far
NIH funded 5 projects under PAR-22-030 in fiscal 2023 to 2026, at 4 institutions. The median yearly award was $1.5 million.
| Project | Institution | Fiscal year | Award |
|---|---|---|---|
| Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS | Columbia University Health Sciences | 2026 | $5.2 million |
| IND-enabling studies of non-immunogenic gene therapy for ultra-rare DMD patients excluded from dystrophin trials | University of Pennsylvania | 2026 | $2.8 million |
| IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial | UT Southwestern Medical Center | 2025 | $744,158 |
| Viral Gene Therapy for Menkes Disease | Columbia University Health Sciences | 2025 | $2.5 million |
| Advancement of Prion Protein-Lowering Divalent siRNA Therapy for Prion Disease | Broad Institute, Inc. | 2024 | $806,536 |
Who has won Extramural Research Programs in the Neurosciences and Neurological Disorders grants before
From fiscal 2021 to 2025, Extramural Research Programs in the Neurosciences and Neurological Disorders (93.853) awarded $9.1 billion across 8,641 grant awards, an average of $1.0 million per award.
| Recipient | State | Awards | Amount |
|---|---|---|---|
| The General Hospital Corporation | Massachusetts | 203 | $288 million |
| The Regents of the University of California, San Francisco | California | 248 | $277 million |
| The Johns Hopkins University | Maryland | 230 | $276 million |
| The Trustees of Columbia University in the City of New York | New York | 188 | $226 million |
| Regents of the University of Michigan | Michigan | 179 | $221 million |
Applying now
This notice no longer takes applications. Apply under the newer notice, PAR-25-327, 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 PAR-22-030 still open?
No. It closed on October 11, 2024. A newer notice is open: PAR-25-327, due dates in the notice, expires October 8, 2027.
Who can apply for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?
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 Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?
Closed October 11, 2024. Applications are no longer accepted; the program's newer notices are on this page when there are any.
Who has been funded under PAR-22-030?
NIH RePORTER lists 5 projects funded under PAR-22-030 in fiscal 2023 to 2026, at 4 institutions. The most were at Columbia University Health Sciences, University of Pennsylvania, UT Southwestern Medical Center.
Who has won Extramural Research Programs in the Neurosciences and Neurological Disorders grants before?
The largest recipients over five fiscal years were The General Hospital Corporation, The Regents of the University of California, San Francisco, The Johns Hopkins University. The program made 8,641 awards worth $9.1 billion in that time.
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