etx101

ETX101 is an investigational gene therapy developed by Encoded Therapeutics for the treatment of Dravet syndrome caused by mutations in the SCN1A gene. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a transgene encoding an engineered transcription factor specifically designed to upregulate endogenous SCN1A expression in GABAergic inhibitory neurons. This targeted approach aims to restore NaV1.1 sodium channel function and address the underlying genetic cause of Dravet syndrome with a single intracerebroventricular administration. Preclinical studies have demonstrated increased NaV1.1 protein expression, prolonged survival, and reduced seizure frequency in animal models[1][2][5][7]. ETX101 has received Orphan Drug Designation and Rare Pediatric Disease Designation from regulatory agencies in both the US and EU[4][5][8].

Other names

Dravet syndrome viral gene therapy - Encoded Therapeutics