Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)
ID: 336476Type: 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/336476

Additional Information

http://grants.nih.gov/grants/guide/pa-files/PAR-22-030.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 (U01 - Clinical Trial Optional)." This initiative aims to support Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based therapies targeting ultra-rare neurological and neuromuscular disorders, with a focus on developing promising clinical candidates backed by robust biological rationale and proof of concept data. The program is particularly significant as it addresses the unmet medical needs of approximately 95% of rare diseases that currently lack effective treatments, emphasizing collaboration with NIH staff and access to resources for successful applicants. Interested parties must submit their applications by October 11, 2024, and can reach out to the NIH OER Webmaster at OERWebmaster03@od.nih.gov for further inquiries. Additional details can be found at the provided link: http://grants.nih.gov/grants/guide/pa-files/PAR-22-030.html.

    Point(s) of Contact
    OERWebmaster03@od.nih.gov
    Files
    Title
    Posted
    PAR-22-030-Full-Announcement.html
    PAR-22-030.html
    The Department of Health and Human Services, through the National Institutes of Health (NIH), has issued a funding opportunity aimed at advancing gene-based therapies for ultra-rare neurological and neuromuscular disorders. The initiative supports Investigational New Drug (IND)-enabling studies to prepare therapies for clinical trials. Key objectives include developing suitable therapeutic candidates, conducting preclinical studies, and assembling IND applications for FDA review. The program emphasizes collaboration with NIH staff and resource access for successful applicants, facilitating knowledge-sharing and technical support. Eligible applicants include higher education institutions, non-profit organizations, and government entities, aiming to tackle the unmet medical needs of estimated 95% of rare diseases lacking effective treatments. Emphasis is placed on projects with robust proof-of-concept data and clinical therapeutic candidates targeting specific pathological variants. Applications are due by designated deadlines, and successful projects may receive further funding for First-in-Human clinical trials. The funding opportunity reflects the NIH's commitment to supporting innovative solutions for rare disorders through cooperative agreements, ensuring applicant adherence to strict NIH guidelines and the potential for significant scientific advancements.
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