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 federal opportunity · PAR-25-327
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health lists this opportunity for nonprofit applicant type listed. Review the coded eligibility, award range, source synopsis, and official notice before deciding whether to apply.
Health
Deadline
October 8, 2027
352 days remaining
Award range
—
official range may be incomplete
Applicant signal
Nonprofit applicant type listed
the official notice controls eligibility
Coded applicant types
- 00 — State governments
- 01 — County governments
- 02 — City or township governments
- 04 — Special district governments
- 05 — Independent school districts
- 06 — Public and state-controlled institutions of higher education
- 07 — Federally recognized Native American tribal governments
- 08 — Public housing authorities and Indian housing authorities
- 11 — Native American tribal organizations other than federally recognized tribal governments
- 12 — 501(c)(3) nonprofits other than institutions of higher education
- 13 — Nonprofits without 501(c)(3) status other than institutions of higher education
- 20 — Private institutions of higher education
- 22 — For-profit organizations other than small businesses
- 23 — Small businesses
- 25 — Other applicants; see the official eligibility clarification
These are high-level Grants.gov codes. Additional eligibility text, geographic restrictions, and program-specific rules in the official notice can narrow them.
Before investing application time
- Confirm entity eligibility in the full Notice of Funding Opportunity.
- Check SAM.gov and Grants.gov registration requirements and timing.
- Read every attachment for matching, cost-share, geography, and award conditions.
- Verify the exact deadline time and any agency-specific submission portal.
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Source: Grants.gov daily XML extract, 2026-10-21 extract. This page reproduces selected public listing fields for research convenience. The official Notice of Funding Opportunity and agency instructions control. This product uses the Grants.gov API but is not endorsed or certified by the U.S. Department of Health and Human Services.