Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)

The National Institutes of Health (NIH) is seeking applications for the Ultra-Rare Gene-Based Therapy (URGenT) network to support Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics 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. • The opportunity is open to various applicant types, including state governments, public housing authorities, special district governments, public and state-controlled institutions of higher education, for-profit organizations, non-profits, and small businesses. • The funding instrument for this opportunity is a Cooperative Agreement. • The funding activity category is Health. • The expected application count is 100, and the expected application size is 0. • The opportunity is electronic and requires the use of the eRA Service Desk. • The competition title is "Use for due dates on or after January 25, 2025."