Scout | PAR-25-327: Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01

grant PAR-25-327

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

Summary

This grant opportunity aims to support projects that speed up the development of a strong clinical candidate with solid scientific evidence and proven success in a relevant model system for a specific patient group. The ultimate goal is to reach the stage of filing an Investigational New Drug (IND) application and starting a clinical trial.

This Notice Of Funding Opportunity (NOFO) is part of the NINDS Ultra-Rare Gene-based Therapy (URGenT) network. The mission of URGenT is to support and facilitate the development of gene-based or transcript-directed therapeutic agents with compelling proof-of-concept (POC) data towards First-in-H…

Funding Source

Agency: National Institutes of Health

Sub-agency: National Institute of Neurological Disorders and Stroke

Eligibility

Higher Education Institutions – Private Institutions of Higher Education

Nonprofit Organizations – without 501(c)(3) IRS Status (other than Institutions of Higher Education)

Small Businesses

Native American Tribal Organizations (other than Federally recognized tribal governments)

Federal Government – Eligible Agencies of the Federal Government

U.S. Territory or Possession

Independent School Districts

Public Housing Authorities/Indian Housing Authorities

Faith-based or Community-based Organizations

Regional Organizations

Foreign/Non-domestic eligibility: 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 within applicat…

Dates

Opportunity Released: December 18, 2024

Supporting links

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