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ACCEPTING APPLICATIONS · COOPERATIVE AGREEMENT

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

National Institutes of Health · Health

Accepting applications. The agency is taking applications now through Grants.gov.
Deadline
Oct 8, 2027 (349 days left)
Who can apply
Others
Award range
Not stated
Expected awards
Not stated
Total program funding
Not stated
Your own money required
No cost sharing or matching required
Opportunity number
PAR-25-327
Posted
Dec 18, 2024
Assistance listing
93.853

More on who can apply

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 Pos

What it 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.

Open the official notice on Grants.gov ↗

What to do next

  1. Open the official notice on Grants.gov and download the full announcement (the NOFO) and any attachments.
  2. Check you can apply: the eligible applicants above, and any detail in the notice's eligibility section.
  3. Register your organization in SAM.gov (choose "all awards") and create a Grants.gov account; allow several weeks the first time.
  4. Apply through Grants.gov exactly as the notice says, before Oct 8, 2027. Late applications are usually not accepted.

New to this? Applying for a federal grant, step by step · Register to apply · How to read the notice · Write the narrative · Build the budget · Agency contact: grantsinfo@nih.gov

From Grants.gov's official daily extract of Oct 25, 2026. The notice on Grants.gov and its attachments are what count. UX Contract is independent and not a government website.

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