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

National Institutes of HealthAmount not specifiedDue Oct 8, 2027

Researchers Schools & universities Government / tribal Nonprofits Narrow eligibility

Deadline: Oct 8, 2027
Posted: Dec 18, 2024
Award amount: Amount not specified
Focus areas: Health

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In plain English

This NIH/NINDS U01 funds IND-enabling studies and planning for first-in-human testing of gene-based therapies for ultra-rare neurological and neuromuscular disorders. Eligible applicants are research and education institutions, including minority-serving and tribal-serving institutions, faith- or community-based organizations, and tribal governments; foreign organizations are not eligible, and it targets research organizations rather than individuals. No award amount is specified. The ultra-rare gene-therapy focus makes eligibility narrow.

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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.

About this grant

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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Source: public records via Grants.gov. UseGrants is an independent aggregator, not affiliated with any funding agency. Always confirm details on the official listing before applying.