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

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

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.

Source

Agency: National Institutes of Health

Sub-agency: National Institute of Neurological Disorders and Stroke

Dates

Opportunity Released: December 18, 2024

Eligibility

government

individual

industry:manufacturing

industry:research

nonprofit

small_business

tribal

university

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

Keywords

Gene therapy

Ultra-rare diseases

Clinical candidates

Biological rationale

Proof of concept

IND filing

Clinical trial initiation

Patient population

Model system

Funding opportunity

Supporting Links

Documents

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