National Institutes of Health

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

PAR-25-327GrantGrant491 days leftGrants.gov

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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.

Submission Details

Response Deadline: Oct 8, 2027

Details

Type: Grant
Deadline: Oct 8, 2027
Source: Grants.gov
First Seen: Mar 1, 2026

Point of Contact

National Institutes of Health

[email protected]301-402-2541

Secondary POC

NIH Grants Information [email protected]

View on Grants.gov

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

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