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: Small businesses, Native American tribal organizations (other than Federally recognized tribal governments), Special district governments, Public and State controlled institutions of higher education, Independent school districts, Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education, City or township governments, For profit organizations other than small businesses, State governments, Public housing authorities/Indian housing authorities, Native American tribal governments (Federally recognized), Others (see text field entitled "Additional Information on Eligibility" for clarification), County governments, Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education, Private institutions of higher education
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.