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

Open

National Institutes of Health · Opportunity number PAR-25-327

PostedDec 18, 2024
Closing dateOct 8, 2027
Award ceilingNot listed
Expected awardsNot listed
From Grants.gov

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 fundingNot listed
Award floorNot listed
Award ceilingNot listed
CategoryHealth
Assistance listing93.853,

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.