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.
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.
Related Programs
Extramural Research Programs in the Neurosciences and Neurological Disorders
Department of Health and Human Services
Agency: Department of Health and Human Services
Office: National Institutes of Health
Estimated Funding: $80,000,000
Office: National Institutes of Health
Estimated Funding: $80,000,000
Who's Eligible
Relevant Nonprofit Program Categories
Obtain Full Opportunity Text:
https://grants.nih.gov/grants/guide/pa-files/PAR-25-327.html
Additional Information of Eligibility:
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.
Full Opportunity Web Address:
http://grants.nih.gov/grants/guide/pa-files/PAR-25-327.html
Contact:
Agency Email Description:
See Section VII. Agency Contacts within the full opportunity announcement for all other inquires.
Agency Email:
Date Posted:
2024-12-18
Application Due Date:
Archive Date:
2027-11-13
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