Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Open Posted
- Opportunity number
- PAR-25-327
- Agency
-
National Institutes of Health
Department of Health and Human Services
- Applications due
- Award size
- Not on record
Opportunity facts
- Assistance listings
- Funding instrument
- Cooperative Agreement
- Category
-
- Health
- Eligible applicants
-
- City/Township Governments
- For Profit Organizations
- Small Businesses
- Other
- Higher Education Inst. (Private)
- Independent School Districts
- County Governments
- Special District Governments
- Public or Indian Housing Auth.
- Non-501(c)(3) Nonprofits
- State Governments
- 501(c)(3) Nonprofits
- Higher Education Inst. (Public)
- Tribal Governments (Federally Recognized)
- Tribal Organizations (Not Federally Recognized)
- Points of contact
-
- NIH OER Webmaster grantsinfo@nih.gov
- Full announcement
Opportunity details come from Grants.gov. Updated .
About this opportunity
The National Institutes of Health is soliciting applications for the Ultra-Rare Gene-Based Therapy (URGenT) network to fund Investigational New Drug-enabling studies and planning activities for First-in-Human clinical testing of gene-based or transcript-directed therapeutics for ultra-rare neurological or neuromuscular disorders, with applications due October 8, 2027.
Eligible applicants span a broad range of organizations: state and local governments, tribal governments and organizations (federally and non-federally recognized), public and private higher education institutions, 501(c)(3) and non-501(c)(3) nonprofits, for-profit organizations, small businesses, and other entity types including faith-based and community-based organizations, HBCUs, HSIs, AANAPISIs, TCCUs, and U.S. territories. Non-domestic (non-U.S.) entities and non-domestic components of U.S. organizations are not eligible; foreign components as defined in the NIH Grants Policy Statement are allowed.
The program supports development of clinical candidates targeting ultra-rare disorders through oligonucleotides and viral-based gene therapies, emphasizing those with robust biological rationale and demonstrated proof-of-concept data in model systems relevant to a specified patient population. Applicants must advance promising candidates toward Investigational New Drug filing and clinical trial initiation.
Awards will be issued as U01 cooperative agreements under CFDA 93.853 (Extramural Research Programs in the Neurosciences and Neurological Disorders). The source does not specify award ceilings, award floors, expected number of awards, period of performance, or cost share requirements.
Synopsis
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.
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.
Files
| File | Type | Posted |
|---|---|---|
| PAR-25-327-Full-Announcement.html | HTML file | |
| PKG00289741.pdf |
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