- Conditions
- Tay-Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose
- Biological
- Lead sponsor
- Terence Flotte
- Other
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Worcester, Massachusetts
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