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Federal Open GRANTS-GOV-357817

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

National Institutes of Health (NIH) · Competitive · Science & Technology

At a glance

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional) is a federal competitive grant from National Institutes of Health for the science & technology sector. Eligible applicants: Special district governments; Public and State controlled institutions of higher education; State governments; Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education; Others (see t. The application deadline is October 8, 2027. Apply via the official listing at www.grants.gov.

Amount
See listing
Deadline
Oct 8, 2027
Type
Competitive
Status
Open

About this program

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.

Who can apply

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

How to apply

Apply through Grants.gov using funding opportunity number PAR-25-327. Register your organization (SAM.gov + Grants.gov) before the deadline, then submit the application package listed in the official notice.

Apply at official source