Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01, Clinical Trial Not Allowed)
ID: 336489Type: Posted
Overview

Buyer

National Institutes of Health (HHS-NIH11)

Eligible Applicants

Others

Funding Category

Health

Funding Instrument

Grant

Opportunity Category

Discretionary

Cost Sharing or Matching Requirement

Yes

Original Source

https://grants.gov/search-results-detail/336489

Additional Information

http://grants.nih.gov/grants/guide/pa-files/PAR-22-028.html
Timeline
    Description

    The National Institutes of Health (NIH) is offering a funding opportunity titled "Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01)," aimed at supporting researchers in the development of gene-based therapies for ultra-rare neurological disorders. This initiative provides access to contract research organizations (CROs) and subject matter experts (SMEs) to facilitate planning, manufacturing, and limited nonclinical development efforts necessary for investigational new drug (IND) submissions. The program is critical for advancing therapeutic candidates that have shown proof of concept, with a focus on bridging the gap between research and practical treatment solutions for patients with urgent medical needs. Interested applicants must submit their proposals by January 24, 2025, and can find additional information and application details at the NIH grants website or contact the NIH OER Webmaster at OERWebmaster03@od.nih.gov.

    Point(s) of Contact
    OERWebmaster03@od.nih.gov
    Files
    Title
    Posted
    PAR-22-028-Full-Announcement.html
    PAR-22-028.html
    The Department of Health and Human Services is offering the Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access (X01), a funding opportunity facilitated by the National Institutes of Health (NIH) and specifically the National Institute of Neurological Disorders and Stroke (NINDS). This initiative aims to provide researchers with access to contract research organizations (CROs) and subject matter experts (SMEs) to assist in the development of gene-based therapies for ultra-rare neurological disorders. The focus is on supporting planning, manufacturing, and limited nonclinical development efforts necessary for investigational new drug (IND) submissions, which are crucial for progressing toward clinical trials. Researchers are encouraged to propose therapeutic candidates that have demonstrated proof of concept through rigorous experimentation, targeting specific ultra-rare genetic diseases. Key activities allowed include forming project teams, conducting feasibility assessments, manufacturing process development, and regulatory strategy formation, all within a maximum project period of two years. The application process is structured to comply with NIH grants policies, requiring participation from eligible institutions ranging from higher education to nonprofit and for-profit entities. The program emphasizes rapid therapeutic advancements for patients with urgent medical needs and aims to bridge the gap between research and practical treatment solutions.
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