Gene therapy trial aims to tame severe seizures in young children
Disease control
Recruiting now
This study tests a gene therapy called ETX101 in infants and children with Dravet syndrome, a severe genetic epilepsy. The treatment uses a harmless virus to deliver a working copy of the SCN1A gene to brain cells, aiming to reduce seizures and improve development. The trial incl…
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated May 20, 2026 11:56 UTC