Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
- Funder
- National Institutes of Health
- Award amount
- Not specified
- Deadline
- October 8, 2027
- Eligible countries
- United States
Summary
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.
Summary as listed on Grants.gov (opportunity 357817). Always read the full announcement for eligibility, limits and how to apply.
Read the full announcement on Grants.gov →What National Institutes of Health reviewers look for
- Importance of the research (significance and innovation), scored 1–9.
- Rigor and feasibility of the approach, scored 1–9. Preliminary data and alternative strategies carry much of this.
- Expertise and resources, rated sufficient or not, without a score.
- Overall impact: the likelihood the project will have a sustained, powerful influence on its field.
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