Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
ID: 357817Type: Posted
Overview

Buyer

National Institutes of Health (HHS-NIH11)

Eligible Applicants

Others

Funding Category

Health

Funding Instrument

Cooperative Agreement

Opportunity Category

Discretionary

Cost Sharing or Matching Requirement

Yes

Original Source

https://grants.gov/search-results-detail/357817

Additional Information

http://grants.nih.gov/grants/guide/pa-files/PAR-25-327.html
Timeline
    Description

    The National Institutes of Health (NIH) is offering a funding opportunity titled "Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders" (PAR-25-327), aimed at supporting Investigational New Drug (IND)-enabling studies and preparations for First-in-Human (FIH) clinical trials. This initiative focuses on advancing innovative gene-based therapies, such as oligonucleotides and viral vector approaches, for ultra-rare disorders affecting fewer than 6,000 patients, with an emphasis on projects that demonstrate robust proof of concept data and readiness for clinical trials. Eligible applicants include a wide range of organizations, including higher education institutions and various nonprofit and for-profit entities, with applications requiring detailed project planning and compliance with Good Manufacturing Practices (GMP). The application period opens on January 9, 2025, with a close date of October 8, 2027, and interested parties can reach out to NIH Grants Information at grantsinfo@nih.gov for further inquiries.

    Point(s) of Contact
    grantsinfo@nih.gov
    Files
    Title
    Posted
    PAR-25-327-Full-Announcement.html
    PAR-25-327.html
    The National Institutes of Health (NIH), through the National Institute of Neurological Disorders and Stroke (NINDS), announces a funding opportunity titled "Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders" (PAR-25-327). The program aims to fund research for Investigational New Drug (IND)-enabling studies and preparation for First-in-Human (FIH) trials focusing on gene-based therapies for ultra-rare diseases affecting fewer than 6,000 patients. A milestone-driven cooperative agreement mechanism (U01) will be utilized, supporting projects that demonstrate robust proof of concept data and are poised for clinical trial readiness. Key funding goals include advancing innovative therapies such as oligonucleotides and viral vector-based approaches while facilitating regulatory pathways to expedite patient access to treatments. Eligible applicants include higher education institutions and various nonprofit and for-profit organizations. Applications require detailed project planning, including compliance with Good Manufacturing Practices (GMP) and submission of IND applications. Important deadlines include an application opening date of January 9, 2025, with funding decisions following rigorous peer review based on scientific merit and relevance to program priorities. The opportunity underlines NIH's commitment to supporting groundbreaking research in the field of ultra-rare neurological and neuromuscular disorders.
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