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 towards an IND filing and the initiation of a clinical trial.
Eligibility
- City Or Township Governments
- For Profit Organizations Other Than Small Businesses
- Small Businesses
- Other
- Private Institutions of Higher Education
- Independent School Districts
- County Governments
- Special District Governments
- Public and Indian Housing Authorities
- Nonprofits Non Higher Education Without 501c3
- State Governments
- Nonprofits Having A 501(c)(3) Status With the Irs Other Than Institutions of Higher Education
- Public and State Institutions of Higher Education
- Native American Tribal Governments Federally Recognized
- Native American Tribal Organizations Other Than Federally Recognized Tribal Governments
Funding categories
- Health
Official source and attribution
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