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

Federal funding opportunity PAR-25-327 from National Institutes of Health (Department of Health and Human Services).

Apply on Grants.gov →Application closes October 8, 2027

Posted
December 18, 2024
Closes
October 8, 2027
Cost sharing
No
Instrument
Cooperative Agreement
Assistance listing
93.853
Category
Health
Archives
November 13, 2027

Program funding history

Awards made under Assistance Listing 93.853 across FY2024–FY2026, from public federal spending records.

FY2024 obligated
$1.8B
FY2025 obligated
$1.8B
FY2026 (to date) obligated
$1B
Awards in window
9,367

Top recipients: The General Hospital Corporation, Regents of the University of California, San Francisco, the, The Johns Hopkins University, Regents of the University of Michigan, The Trustees of Columbia University in the City of New York

Source: USAspending.gov · refreshed July 2026

Synopsis

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

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.

How to apply

Applications go through the official government listing. Grants Radar links you straight to the source.

View on Grants.gov   Full announcement

Agency contact: National Institutes of Health · grantsinfo@nih.gov · 301-402-2541

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