Experimental gene therapy targets rare fatal brain diseases in kids
Disease control
Terminated
This study tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that damage the brain. The therapy aimed to deliver healthy genes to brain cells to restore a missing enzyme. The trial enrolled up to 9 children a…
Phase: PHASE1 • Sponsor: Terence Flotte • Aim: Disease control
Last updated May 12, 2026 13:41 UTC