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

US Dept. of Health & Human Services: National Institutes of Health (NIH)

Next predicted deadline: Oct 10, 2023 (Full proposal)

Later predicted deadlines: Jan 8, 2024 (Letter of inquiry), Feb 8, 2024 (Full proposal), May 7, 2024 (Letter of inquiry), Jun 7, 2024 (Full proposal)

Grant amount: Unspecified amount

Funding uses: Research

Location of project: United States

Location of residency: United States

Overview:

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 funding opportunity announcement (FOA) 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.

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