Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is offered by National Institutes of Health. Max award: Not specified. Applications close on Oct 8, 2027.
| Opportunity number | PAR-25-327 |
|---|---|
| Agency | National Institutes of Health |
| Status | Posted |
| Posted | Dec 18, 2024 |
| Closes | Oct 8, 2027 |
| Award ceiling | Not specified |
| Award floor | Not specified |
| Total program funding | Not specified |
| Expected awards | Not specified |
| Who can apply | Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, Public and State controlled institutions of higher education, Private institutions of higher education, Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), State governments, Independent school districts, For profit organizations other than small businesses, Small businesses, City or township governments, Others (see text field entitled "Additional Information on Eligibility" for clarification), Native American tribal governments (Federally recognized), Special district governments, County governments |
| Category | Health |
Summary (excerpt)
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 bio…
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