Encoded Therapeutics to Present New Data on ETX101 Gene Therapy for Dravet Syndrome at the 16th European Epilepsy Congress
– ETX101 is an AAV9-based gene regulation therapy being developed as a one-time, disease-modifying treatment for SCN1A+
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Encoded Therapeutics Inc., a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that new results from the POLARIS clinical program of ETX101, its investigational AAV9-based gene regulation therapy for SCN1A+ Dravet syndrome, will be featured in a platform presentation at the 16th European Epilepsy Congress on September 7, 2026, in Athens, Greece.
Professor Ingrid Scheffer, Pediatric Neurologist and Laureate Professor of Pediatric Neurology at The University of Melbourne, will present up to 117 weeks of follow-up data, including substantial and sustained seizure reductions and clinically meaningful neurodevelopmental gains following a single dose of ETX101 in children living with Dravet syndrome.
Presentation Details
Title: Interim Safety and Preliminary Efficacy of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome: Results from the POLARIS Phase 1/2 Program
Platform Session: Drug Therapy and Clinical Trials
Presenter: Professor Ingrid Scheffer, MBBS, PhD, FAES, FRS, Pediatric Neurologist and Laureate Professor of Pediatric Neurology at The University of Melbourne
Location: Banqueting Hall, Upper Level, Megaron Athens International Conference Centre
Date and Time: Monday, September 7, 2026; 1:02 PM – 1:11 PM EEST (UTC+3)
About ETX101
ETX101 is an investigational AAV9-based gene regulation therapy designed to increase the expression of the SCN1A gene to restore sodium channel function in inhibitory interneurons. By targeting the root mechanism, ETX101 has the potential to treat the full spectrum of Dravet syndrome symptoms, including seizures, communication and cognitive impairment, behavioral issues, and motor dysfunction. The therapy is administered via a single intracerebroventricular (ICV) injection and is designed for long-term benefit. ETX101 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations from the FDA. It also was selected for the FDA’s CMC Development and Readiness Pilot (CDRP) program and received Orphan designation from the European Medicines Agency (EMA).
About Encoded Therapeutics
Encoded Therapeutics is a clinical-stage biotechnology company developing one-time precision genetic medicines for severe monogenic and common neurological disorders. The company’s vector engineering platform enables highly-targeted and cell-type-selective control of gene expression in the brain and peripheral nervous system, allowing potent and precise modulation of disease-relevant genes to address underlying disease biology. Encoded’s end-to-end innovation engine—spanning discovery, development, and in-house GMP manufacturing—creates a streamlined path to advance a diversified pipeline of one-time treatments across a broad range of neurological conditions. Encoded is driven by a mission to meaningfully improve the lives of patients and families affected by devastating neurological disorders. For more information, please visit www.encoded.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260902159769/en/
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