Funder: National Institutes of Health (NIH)
Due Dates: October 10, 2024 (New & Renewal) | Rolling (Late submissions considered case-by-case)
Funding Amounts: Budgets are not limited but must reflect actual project needs; typical project period up to 2 years.
Summary: Supports IND-enabling studies and clinical trial planning to advance gene-based therapies for ultra-rare neurological and neuromuscular disorders toward first-in-human trials.
Key Information: Cooperative agreement (U01) mechanism with substantial NIH involvement; clinical trial optional but limited to mechanistic or basic experimental studies with humans; excludes efficacy or safety trials.