Grant opportunity · PAR-25-327
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Who can apply (as listed)
- For-profit organizations (other than small businesses)
- Special district governments
- Native American tribal organizations (other than federally recognized governments)
- Nonprofits without 501(c)(3) status (other than institutions of higher education)
- Native American tribal governments (federally recognized)
- Others (see the opportunity text)
- Small businesses
- City or township governments
- Public and state-controlled institutions of higher education
- Public housing authorities / Indian housing authorities
- County governments
- State governments
- Independent school districts
- Nonprofits with 501(c)(3) status (other than institutions of higher education)
- Private institutions of higher education
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.
Excerpt as published. The full synopsis, required forms, and any amendments are on the official listing.
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