health · HHS-NIH11

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

National Institutes of Health · PAR-25-327

412 days left

Deadline

October 8, 2027

GRANTQUICK SUMMARYPlain-English Overview

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional). National Institutes of Health. 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... Funding amounts not specified.

Who Should Apply

City/township governments, For-profit organizations, Small businesses, Other eligible entities, Private universities/colleges (+10 more)

Who Should NOT Apply

Organizations not matching the eligible applicant types listed above

Key Requirements (Plain English)

•Other Eligible Applicants include the following: Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Is...
•Deadline: 2027-10-08

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Focus your proposal on clear outcomes, alignment with agency priorities, and demonstrate organizational capacity.

Competitiveness: Moderate to high — federal grants are always competitive

What This Grant Funds

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.

Attachments

PDF

PAR-25-327 Full Announcement Text

PAR-25-327-Full-Announcement.html

Who Can Apply

Other Eligible Applicants include the following: Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Eligible Agencies of the Federal Government; Faith-based or Community-based Organizations; Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Indian/Native American Tribal Governments (Other than Federally Recognized); Non-domestic (non-U.S.) Entities (Foreign Organizations); Regional Organizations; Tribally Controlled Colleges and Universities (TCCUs) ; U.S. Territory or Possession; Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply. Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply. Foreign components, as defined in the NIH Grants Policy Statement, are allowed.

Eligible Applicant Types

City/Township GovernmentsFor-Profit OrganizationsSmall BusinessesOther (see NOFO for details)Private Colleges & UniversitiesIndependent School DistrictsCounty GovernmentsSpecial District GovernmentsPublic And Indian Housing AuthoritiesNonprofits without 501(c)(3)State Governments501(c)(3) NonprofitsPublic Colleges & UniversitiesFederally Recognized Tribal GovernmentsOther Tribal Organizations

Funding Details

Cost Sharing Required?: No
Funding Instrument: cooperative_agreement

Key Dates

Posted: December 18, 2024

Application Deadline: October 8, 2027 (412 days remaining)

Agency Contact

NIH Grants Information grantsinfo@nih.gov

grantsinfo@nih.gov

Ready to apply?

View the full NOFO and submit your application on Grants.gov

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