Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
National Institutes of Health
Award ceiling
—Award floor
—Posted
Dec 18, 2024Closes
Oct 8, 2027Location eligibility
No specific state restriction found in this listing — check the full opportunity for details.Categories: Health
Eligible applicants: City or township governments, Public and State controlled institutions of higher education, Native American tribal organizations (other than Federally recognized tribal governments), For profit organizations other than small businesses, Private institutions of higher education, Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education, Others (see text field entitled "Additional Information on Eligibility" for clarification), State governments, Public housing authorities/Indian housing authorities, Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, Special district governments, Native American tribal governments (Federally recognized), Small businesses, County governments, Independent school districts
Description
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.