Funder: National Institutes of Health
Due Dates: June 9, 2025 (New/Renewal/Resubmission) | October 9, 2025 | February 9, 2026 | June 9, 2026 | October 9, 2026 | February 9, 2027 | June 9, 2027 | October 8, 2027
Funding Amounts: No budget cap; project period up to 3 years; budgets must reflect actual needs.
Summary: Supports IND-enabling studies and clinical trial planning for gene-based therapies targeting ultra-rare neurological and neuromuscular disorders.
Key Information: Only mechanistic clinical trials are allowed; safety/efficacy trials are not supported under this mechanism.