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

National Institutes of Health · United States government procurement

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October 08, 2027
Response Due
Active
Status

Opportunity Overview

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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Solicitation Details

Issuing agencyNational Institutes of Health
CountryUnited States
CategoryHospital Construction
PublishedDecember 18, 2024
Procurement stageRequest for Proposal (RFP)
Response dueOctober 08, 2027
StatusOpen — accepting responses
Official sourceView original notice
Last verifiedOctober 11, 2026

Source: U.S. Government (Simpler.Grants.gov) — public domain. Not affiliated with or endorsed by the United States Government.

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