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.

Program Details

National Institutes of Health is a federal agency that offers the Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) grant program to support eligible programs and projects. This funding opportunity is available nationally and is administered by National Institutes of Health through Grants.gov. Organizations seeking funding for eligible programs and projects should review the details below and confirm their eligibility before preparing an application.

Eligibility

Eligible applicants include government agencies and municipalities, eligible organizations, educational institutions, and nonprofit organizations. Organizations that meet the eligibility criteria are encouraged to apply and to review the full program guidelines before submission. Because eligibility rules and funding priorities can change between cycles, applicants should always confirm the current requirements with National Institutes of Health or on Grants.gov before investing time in an application.

Funding and Timeline

The application deadline is October 8, 2027 (437 days remaining). Applicants should allow sufficient time to gather required documentation, including organizational information, project descriptions, budgets, and supporting materials as specified in the program guidelines. Submitting a complete application well ahead of the deadline reduces the risk of disqualification on technical or administrative grounds.

Program Focus

This grant supports eligible programs and projects. This grant is associated with NAICS industry classifications: 541714, 621111, 621112, 622. Organizations operating in these sectors are encouraged to review eligibility. Funded projects are expected to demonstrate measurable outcomes aligned with the program's stated objectives. Grant recipients may be required to submit progress reports and final evaluations as a condition of the award, and to retain records for audit and compliance purposes.

How to Apply

Eligible organizations can apply through Grants.gov. Create a free organization profile on GrantRegister to see whether this grant matches your organization's NAICS code, entity type, location, and focus area, and to receive a weekly digest of every open grant you qualify for.

GrantRegister maintains a complete, authoritative registry of US federal, state, and foundation grant opportunities — matched automatically to your organization profile and delivered every Monday.

Eligibility

• Funded by NIH

Award details

Applications close on October 8, 2027 (437 days remaining).

Categories

Not categorized