Product

AXO-AAV-GM2

Aliases

AAVrh8-HEXA and AAVrh8-HEXB

1 clinical trial

2 indications

Indication

Tay-Sachs Disease

Indication

Sandhoff disease

Clinical trial

A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Status: Active (not recruiting), Estimated PCD: 2024-11-01