This grant, offered by the Ultra-Rare Gene-Based Therapy (URGenT) network, is designed to support the crucial Investigational New Drug (IND)-enabling studies and planning activities essential for First-in-Human (FIH) clinical testing. It focuses specifically on gene-based or transcript-directed therapeutics, including oligonucleotides and viral-based gene therapies, targeting ultra-rare neurological and neuromuscular disorders. The primary purpose is to significantly accelerate the development of highly promising clinical candidates. Applicants must demonstrate robust biological rationale and strong proof-of-concept (POC) data in relevant model systems. The ultimate aim is to facilitate progress towards successful IND filings and the prompt initiation of clinical trials for these vital treatments.
Opportunity ID: 357817
General Information
| Document Type: | Grants Notice |
| Funding Opportunity Number: | PAR-25-327 |
| Funding Opportunity Title: | Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional) |
| Opportunity Category: | Discretionary |
| Opportunity Category Explanation: | – |
| Funding Instrument Type: | Cooperative Agreement |
| Category of Funding Activity: | Health |
| Category Explanation: | – |
| Expected Number of Awards: | – |
| Assistance Listings: | 93.853 — Extramural Research Programs in the Neurosciences and Neurological Disorders |
| Cost Sharing or Matching Requirement: | No |
| Version: | Synopsis 1 |
| Posted Date: | Dec 18, 2024 |
| Last Updated Date: | Dec 18, 2024 |
| Original Closing Date for Applications: | Oct 08, 2027 |
| Current Closing Date for Applications: | Oct 08, 2027 |
| Archive Date: | Nov 13, 2027 |
| Estimated Total Program Funding: | – |
| Award Ceiling: | – |
| Award Floor: | – |
Eligibility
| Eligible Applicants: | City or township governments County governments For profit organizations other than small businesses Native American tribal organizations (other than Federally recognized tribal governments) Special district governments Native American tribal governments (Federally recognized) Small businesses Private institutions of higher education Others (see text field entitled “Additional Information on Eligibility” for clarification) State governments Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education Public housing authorities/Indian housing authorities Independent school districts Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Public and State controlled institutions of higher education |
| Additional Information on 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 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 Organizations) 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. |
Additional Information
| Agency Name: | National Institutes of Health |
| Description: | 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. |
| Link to Additional Information: | https://grants.nih.gov/grants/guide/pa-files/PAR-25-327.html |
| Grantor Contact Information: | If you have difficulty accessing the full announcement electronically, please contact:
NIH Grants Information
grantsinfo@nih.gov Email:grantsinfo@nih.gov |
Version History
| Version | Modification Description | Updated Date |
|---|---|---|
Related Documents
Folder 357817 Full Announcement-PAR-25-327 -> PAR-25-327-Full-Announcement.pdf
Packages
| Agency Contact Information: | NIH Grants Information grantsinfo@nih.gov Email: grantsinfo@nih.gov |
| Who Can Apply: | Organization Applicants |
| Assistance Listing Number | Competition ID | Competition Title | Opportunity Package ID | Opening Date | Closing Date | Actions |
|---|---|---|---|---|---|---|
| FORMS-I | Use for due dates on or after January 25, 2025 | PKG00289741 | Jan 09, 2025 | Oct 08, 2027 | View |
Package 1
Mandatory forms
357817 RR_SF424_5_0-5.0.pdf
357817 PHS398_CoverPageSupplement_5_0-5.0.pdf
357817 RR_OtherProjectInfo_1_4-1.4.pdf
357817 PerformanceSite_4_0-4.0.pdf
357817 RR_KeyPersonExpanded_4_0-4.0.pdf
357817 PHS398_ResearchPlan_5_0-5.0.pdf
357817 PHSHumanSubjectsAndClinicalTrialsInfo_3_0-3.0.pdf
Optional forms
357817 RR_Budget_3_0-3.0.pdf
357817 RR_SubawardBudget30_3_0-3.0.pdf
357817 PHS398_ModularBudget_1_2-1.2.pdf
357817 PHS_AssignmentRequestForm_4_0-4.0.pdf
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