Grants.govNational Institutes of Health

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

Posted Dec 18, 2024·open

AGENCY

HHS-NIH11

PROGRAM

Discretionary

AMOUNT

—

CLOSES

Oct 8, 2027

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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.

Eligibility

County governments; Public and State controlled institutions of higher education; Native American tribal organizations (other than Federally recognized tribal governments); Private institutions of higher education; City or township governments; Native American tribal governments (Federally recognized); Independent school districts; Public housing authorities/Indian housing authorities; For profit organizations other than small businesses; Others (see text field entitled "Additional Information on Eligibility" for clarification); Small businesses; Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education; State governments; Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education; Special district governments

Contact

National Institutes of Health

grantsinfo@nih.gov

301-402-2541

Additional details

Funding instrument: Cooperative Agreement
Cost sharing required: No
Archived: Nov 13, 2027

Opportunity number: PAR-25-327

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