Translational Efforts to Advance Gene-Based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
Key Information
Due Date: October 8, 2027
Agency: United States Department of Health and Human Services
Source: Federal
Funding Categories:
Funding Amount: Not Specified
Funding Type: Cooperative Agreement
Match Required: No
Status: Posted
Contact Info:
grantsinfo@nih.gov
NIH Grants Information grantsinfo@nih.gov
NIH Grants Information grantsinfo@nih.gov
Overview
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.
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Key Dates
Posted Date: December 18, 2024
Application Due Date: October 8, 2027
Estimated Award Date: Not Specified
Additional Details
Eligible Applicants
- 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/Indian housing authorities
- Native American tribal organizations (other than Federally recognized tribal governments)
- 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
- Private institutions of higher education
- For profit organizations other than small businesses
- Small businesses
- Others