# Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)

Source: https://welltoldgrants.com/grants/par-25-327-translational-efforts-to-advance-gene-based-therapies-for/  
Official notice: https://www.grants.gov/search-results-detail/357817

> National Institutes of Health is offering grants to nonprofits, colleges and universities, state and local governments, tribes, small businesses and businesses. Applications close on October 8, 2027.

| Field | Value |
|---|---|
| Opportunity number | PAR-25-327 |
| Agency | National Institutes of Health |
| Status | Open |
| Deadline | October 8, 2027 |
| Award ceiling | not stated |
| Award floor | not stated |
| Expected awards | not stated |
| Total funding | not stated |
| Cost sharing | not required |
| Assistance Listings | 93.853 |

## Who can apply

- 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 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.

## 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 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.

## Questions people ask

### 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 accepts 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)?

Closes October 8, 2027 (342 days left). Confirm the date and time in the official notice on Grants.gov.