Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
This grant supports studies & planning for early clinical trials of gene-based therapies for ultra-rare neurological & neuromuscular disorders.
Funder: National Institutes of Health
Due Dates: June 9, 2025 (New/Renewal/Resubmission) | October 9, 2025 | February 9, 2026 | June 9, 2026 | October 9, 2026 | February 9, 2027 | June 9, 2027 | October 8, 2027
Funding Amounts: No budget cap; project period up to 3 years; budgets must reflect actual needs.
Summary: Supports IND-enabling studies and clinical trial planning for gene-based therapies targeting ultra-rare neurological and neuromuscular disorders.
Key Information: Only mechanistic clinical trials are allowed; safety/efficacy trials are not supported under this mechanism.
Description
This opportunity, part of the NINDS Ultra-Rare Gene-Based Therapy (URGenT) network, funds translational research to advance gene-based or transcript-directed therapeutics—such as oligonucleotides, viral vectors, gene-modified cell therapies, genome editing, and RNA-targeted small molecules—for ultra-rare neurological and neuromuscular disorders. The program supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing, aiming to accelerate the development of promising clinical candidates with robust biological rationale and proof-of-concept data.
Projects should be ready to complete IND-enabling studies and initiate clinical trial planning. Successful completion may enable subsequent application for FIH clinical trials through URGenT network resources.
