Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
OpenNational Institutes of Health · Opportunity number PAR-25-327
Eligibility
Nonprofits are listed as eligible applicants.
- Private institutions of higher education
- State governments
- Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
- Special district governments
- Native American tribal governments (Federally recognized)
- City or township governments
- For profit organizations other than small businesses
- Public housing authorities/Indian housing authorities
- Small businesses
- Others (see text field entitled "Additional Information on Eligibility" for clarification)
- County governments
- Independent school districts
- Native American tribal organizations (other than Federally recognized tribal governments)
- Public and state controlled institutions of higher education
- Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
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.
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.
Funding
| Total program funding | Not listed |
|---|---|
| Award floor | Not listed |
| Award ceiling | Not listed |
| Category | Health |
| Assistance listing | 93.853, |
Official sources
Last verified on Grants.gov Nov 4, 2026. Grantvello is not affiliated with Grants.gov or National Institutes of Health. The notice on Grants.gov is the binding text.