Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) — National Institutes of Health funding opportunity
National Institutes of Health · Federal agency

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 therapeut...

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Deadline Oct 8 Location Alabama Type grant Level Federal Open posted Dec 18, 2024
✦ AI Summary
  • Who can apply: Federal-level applicants (see eligibility for details).
  • Next deadline: October 8, 2027.
  • Issued by: National Institutes of Health.
How was this generated?

The “key facts” mode pulls structured fields directly from the official source posting (amount, deadline, eligibility tags). The AI mode adds a short plain-English narrative on top, generated from the same source. Always verify with the agency before applying.

AI-generated. Always verify with the official source.

Deadline
Oct 8
Oct 8, 2027

About this opportunity

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.

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Who can apply

Eligibility details aren't on file yet — check the agency source link in the Documents tab for the latest rules.

Geographic eligibility

  • Alabama
  • Alaska
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  • California
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  • Connecticut
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  • District of Columbia

How to apply

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Source documents

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Canonical NOFO, application packet, and forms
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Citation details

Source systemgrants.gov
Source ID357817
PostedDec 18, 2024

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