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

In plain English

National Institutes of Health (Department of Health and Human Services) is accepting applications for this cooperative agreement until Oct 8, 2027 (331 days left).

The listing does not state award amounts or the number of awards.

Eligible applicant types include nonprofits, small businesses, for-profit companies, state governments, local governments, tribal governments & organizations, colleges & universities, school districts and public housing authorities, plus others described in the listing.

The listing says cost sharing or matching is not required.

Built only from the fields in the Grants.gov listing. Always confirm on the official listing.

Browse similar: Nonprofits · Small businesses · For-profit companies · State governments · Local governments · Tribal governments & organizations · Colleges & universities · School districts · Public housing authorities

• 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 with 501(c)(3) status (other than institutions of higher education)
• Nonprofits without 501(c)(3) status (other than institutions of higher education)
• Private institutions of higher education
• For-profit organizations other than small businesses
• Small businesses
• Others (see the eligibility details in the listing)

Additional eligibility information (excerpt from the listing):

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. …

Excerpt from the official synopsis on Grants.gov (shortened; read the full text on the official listing):

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.

Read the full listing on Grants.gov → · Agency's announcement page