Open — closes in 475 days

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

National Institutes of Health Nonprofit eligible

This open federal grant from National Institutes of Health supports health-focused work and is available to nonprofits and other eligible applicants, with applications due Oct 8, 2027.

Open the official Grants.gov listing
Deadline
Oct 8, 2027
Posted
Dec 18, 2024
Opportunity number
PAR-25-327
CFDA
93.853

Opportunity 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.

Who can apply

  • County governments
  • Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
  • Native American tribal governments (Federally recognized)
  • Small businesses
  • Native American tribal organizations (other than Federally recognized tribal governments)
  • Public housing authorities/Indian housing authorities
  • Others (see text field entitled "Additional Information on Eligibility" for clarification)
  • Special district governments
  • For profit organizations other than small businesses
  • State governments
  • Independent school districts
  • Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
  • Public and State controlled institutions of higher education
  • Private institutions of higher education
  • City or township governments

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.

Agency contact

National Institutes of Health

NIH Grants Information grantsinfo@nih.gov

grantsinfo@nih.gov

Ready to apply?

Federal grant applications are submitted through Grants.gov, not VolunteerBadge. Confirm the current deadline and full requirements on the official listing before you begin.

Official Grants.gov listing

We summarize this opportunity from the public Grants.gov database for convenience. It is general information, not legal or grant advice. VolunteerBadge is not affiliated with any agency — rely on the official listing for final details and deadlines.

Need cleared volunteers for your funded program?

VolunteerBadge runs FCRA-compliant, identity-verified background checks for $5 — fast and affordable enough for any grant timeline.

See how it works

Related grant opportunities