A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis — Stella
Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.
(1 site) United States
National Institutes of Health Clinical Center, Bethesda, Maryland
Key details Phase
Early testing / Safety & dosing
Sponsor
National Human Genome Research Institute (NHGRI)
Collaborators
Sio Gene Therapies
Enrollment target
~54 participants
Primary completion
January 2028
Last updated December 2025