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Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health · PAR-25-327 · Deadline: October 8, 2027
Opportunity-exact history — these numbers describe this specific funding opportunity (PAR-25-327), from NIH RePORTER.
to 3 distinct organizations. By FY: 2026: 3
range $650k – $2.5M
Listing summary
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 …
Eligibility: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities, Native American tribal organizations (other), Nonprofits with 501(c)(3), other than higher education, Nonprofits without 501(c)(3), Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others · Categories: Health · official listing ↗
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Sources: NIH RePORTER (opportunity-exact) and USAspending (program-level), labeled per section. GrantsDue computes summaries only; verify anything critical against the originals. Methodology.