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Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
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Service Source Final Application Due Date Funding Available Match Required
Health Care Federal
HHS
10-11-2024 N/A No Match
Required
  • Service
  • Source
  • Final Application Due Date
  • Funding Available
  • Match Required
Status
  • Past
  • Current
  • Forecasted
    • Opportunity Type Discretionary
    • CFDA

      93.853 -- Extramural Research Programs in the Neurosciences and Neurological Disorders

    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 funding opportunity announcement (FOA) 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
    • IHE
    • Local Government
    • Non-Profit
    • Other
    • State Government
    • Tribal Government
    Additional Eligibility Information

    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 Institutions) 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.

    Key Date(s)
    • November 09, 2021: Posted Date
    • December 01, 2023: Last Updated Date
    • October 11, 2024: Current Closing Date for Applications
    • October 11, 2024: Application Due Date
    • November 10, 2024: Application Archive Date
    Contact Information

    NIH OER Webmaster

    OERWebmaster03@od.nih.gov

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