Scientists test gene therapy for devastating childhood brain disease
Disease control
Terminated
This early-stage trial tested a gene therapy called AXO-AAV-GM2 in children with Tay-Sachs or Sandhoff disease, rare and fatal genetic disorders that destroy the brain. The study aimed to see if delivering working copies of the faulty genes directly into the brain and spinal flui…
Phase: PHASE1 • Sponsor: Terence Flotte • Aim: Disease control
Last updated Mar 02, 2026 15:27 UTC