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

This program funds nonclinical, IND-enabling work and clinical trial planning for gene-based or transcript-directed therapies for ultra-rare neurological and neuromuscular disorders, with the goal of preparing an FDA research IND and future first-in-human trial. Eligible applicants include U.S. higher education institutions, nonprofits, for-profit organizations, governments, tribal entities, faith-based and community-based organizations, regional organizations, foreign organizations, and federal agencies, but non-U.S. entities and non-U.S. components of U.S. organizations are not eligible. The award uses a cooperative agreement, budgets are not limited, and the project period must not exceed 3 years. No cost share requirement is stated. Clinical trials are optional, but only mechanistic trials are supported under this announcement; trials focused on safety, efficacy, or effectiveness are not supported here.

✳ AI-generated summary — verify against the announcement

• Health
• Research & Discovery
• Technology & Product Development
• Patients & People with Health Conditions
• Biomedical & Disease Research

• ✓ City or township governments
• ✓ County governments
• ✓ For-profit organizations other than small businesses
• ✓ Independent school districts
• ✓ Native American tribal governments (Federally recognized)
• ✓ Native American tribal organizations (other than Federally recognized)
• ✓ Nonprofits with 501(c)(3) status (other than higher education)
• ✓ Nonprofits without 501(c)(3) status (other than higher education)
• ✓ Others
• ✓ Private institutions of higher education
• ✓ Public and State controlled institutions of higher education
• ✓ Public housing authorities / Indian housing authorities
• ✓ Small businesses
• ✓ Special district governments
• ✓ State governments