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Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
NCT07479953Phase 1Not yet recruiting
- Sponsor
- InUtero Therapeutics
- Start
- Jan 2027
- Primary completion
- Jun 2039
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
01
Overview
Phase 1, open-label, single-arm trial of aav9-glb1 in GM1 gangliosidosis (GM1), sponsored by InUtero Therapeutics. Primary endpoint: Safety at 5 years. Planned start Jan 2027; status: not yet recruiting.
02
Drugs
aav9-glb1
03
Conditions
GM1 gangliosidosis (GM1)
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