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

Updated Oct 20, 2026

Cooperative agreement from the National Institutes of Health, open to small businesses. Closes Oct 8, 2027, 353 days left.

Opportunity number	PAR-25-327
Agency	National Institutes of Health
Funding type	Cooperative agreement
Field	Health
Cost sharing or matching	Not required
Assistance listing	93.853
Posted	Dec 18, 2024
Closes	Oct 8, 2027 (353 days left)
Add the deadline to	Google Calendar

Who can apply

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 and 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 notes)

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.

Full announcement and how to apply on Grants.gov

Health grants right now

There are 350 open federal grants in health that small businesses may apply for; 33 close within 30 days. The median award ceiling is $500,000. The agencies offering the most are National Institutes of Health, Centers for Disease Control and Prevention - ERA and Centers for Disease Control - NCBDDD. Grants in this field stay open a median of 1031 days; this one is open for 1024 days.

Who has won this program before

University Of North Carolina AT Chapel Hill $977.0 million · from Jun 29, 2006
Mayo Clinic $102.2 million · from Sep 15, 2019
The General Hospital Corporation $75.4 million · from Sep 19, 2019
Research Triangle Institute $72.7 million · from Apr 1, 2018
Yale Univ $67.5 million · from Jul 1, 2015

The largest grants and cooperative agreements under assistance listing 93.853 with payments since Oct 1, 2025, from USAspending.gov. Amounts are each award's total.