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

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.

Canonical page: https://grantsonar.com/grants/opportunity/translational-efforts-to-advance-gene-based-therapies-for-ultra-rare-neu-44982ddf-eb05-442e-bdc1-964a8bd6cedb
Source: grants_gov
Source ID: 357817
Last confirmed Oct 13, 2026 · Grants.gov
Deadline: October 8, 2027
Award size: not disclosed

## Answer-first Q&A

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

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) eligibility includes eligible applicants named in the notice. Geographic eligibility is national or notice-specific geography; applicants should confirm the source notice before applying.

### What is the deadline for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) has a listed deadline of October 8, 2027. Grant deadlines can change, so verify the current date with National Institutes of Health before submitting.

### How much funding is available for Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)?

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) lists an award size of not disclosed. Award ranges and total program funding are reported from the source metadata when available.

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

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is funded by National Institutes of Health. GrantSonar tracks the notice with source ID 357817 from grants_gov.