Clinical Trial Update: A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease [Phase 1]

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Summary

Phase 1 dose-escalation and safety/efficacy study of AXO-AAV-GM2 for Tay-Sachs Disease and Sandhoff Disease was terminated, with results posted in May 2025.

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Diseases / indications

• Sandhoff Disease
• Tay-sachs Disease

Drugs mentioned

• AXO-AAV-GM2

Snippet

Sponsor: Terence Flotte | Conditions: Tay-Sachs Disease, Sandhoff Disease | Phase 1 | Status: TERMINATED | Results posted: 2025-05-22