At a glance
This program funds nonclinical, IND-enabling work and clinical trial planning for gene-based or transcript-directed therapies for ultra-rare neurological and neuromuscular disorders, with the goal of preparing an FDA research IND and future first-in-human trial. Eligible applicants include U.S. higher education institutions, nonprofits, for-profit organizations, governments, tribal entities, faith-based and community-based organizations, regional organizations, foreign organizations, and federal agencies, but non-U.S. entities and non-U.S. components of U.S. organizations are not eligible. The award uses a cooperative agreement, budgets are not limited, and the project period must not exceed 3 years. No cost share requirement is stated. Clinical trials are optional, but only mechanistic trials are supported under this announcement; trials focused on safety, efficacy, or effectiveness are not supported here.
What it funds
Official description from grants.gov
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.
Who can apply
- City or township governments
- County governments
- For-profit organizations other than small businesses
- Independent school districts
- Native American tribal governments (Federally recognized)
- Native American tribal organizations (other than Federally recognized)
- Nonprofits with 501(c)(3) status (other than higher education)
- Nonprofits without 501(c)(3) status (other than higher education)
- Others
- Private institutions of higher education
- Public and State controlled institutions of higher education
- Public housing authorities / Indian housing authorities
- Small businesses
- Special district governments
- State governments