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<\/a>\nTreatment with a single dose of ETX101 resulted in a robust and dose-dependent antiseizure effect, with durability through 52 weeks of observation. Clinically meaningful improvements in adaptive behavior were reported across the full age range tested. Notably, children treated before age 2 rapidly diverged from the developmental stagnation expected from natural history, with cognitive trajectories generally consistent with neurotypical development.<\/p>
\n\u201c<\/b>Parents of children with Dravet syndrome live with the fear of every seizure and the heartbreak of watching development stall,\u201d said Mary Anne Meskis, CEO of the Dravet Syndrome Foundation. \u201cTo see the early and robust seizure reductions paired with meaningful developmental gains is profoundly encouraging. Families have been waiting for therapies that don\u2019t just manage symptoms but give their children a chance to keep learning and growing.\u201d<\/p>
\nASGCT Oral Presentation Highlights\n
\nSeizure Reduction:<\/b><\/p> \nNeurodevelopmental Improvements:<\/b><\/p> \n\u201cWatching these young children not only achieve durable seizure reduction but also show early evidence of neurodevelopmental rescue is truly remarkable,\u201d said Sal Rico, M.D., Ph.D., Chief Medical Officer of Encoded. \u201cThese data reinforce our belief that ETX101 has the potential to change the course of the disease and future outlook for the Dravet community.\u201d<\/p> \nTo date, ETX101 has shown a favorable safety profile and has been well-tolerated across all four dose levels, with no treatment- or procedure-related serious adverse events. The most common treatment-related adverse events were transaminase elevations (a known AAV class effect), which were clinically asymptomatic and resolved in all participants.<\/p> \nAbout the POLARIS Clinical Development Program<\/strong><\/p> \nThe POLARIS program is a comprehensive clinical investigation of ETX101 in children and adolescents with SCN1A<\/i>+ Dravet syndrome, comprising multiple Phase 1-3 clinical trials. The first phase of POLARIS includes three ongoing open-label, Phase 1\/2 dose-escalation, multicenter trials (ENDEAVOR Part 1 (US), EXPEDITION (UK), and WAYFINDER (Australia)) in infants and young children aged 6 months to 7 years. ENDEAVOR Part 1B, an expansion study in the US, is actively enrolling children and adolescents aged 4 to 18 years. These studies are evaluating the safety and preliminary efficacy of ETX101 in children and adolescents with Dravet syndrome due to variants in the SCN1A<\/i> gene. The pivotal ENDEAVOR Part 2 study is also ongoing, evaluating seizure and neurodevelopmental outcomes in young children aged 6 months to 4 years.<\/p> \nAbout ETX101<\/strong><\/p> \nETX101 is an investigational AAV9-based gene regulation therapy designed to increase the expression of the SCN1A <\/i>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\u2019s CMC Development and Readiness Pilot (CDRP) Program and received Orphan designation from the European Medicines Agency (EMA).<\/p> \nAbout Encoded Therapeutics<\/strong><\/p> \nEncoded Therapeutics is a clinical-stage biotechnology company developing one-time precision genetic medicines for severe monogenic and common neurological disorders. The company\u2019s 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\u2019s end\u2011to\u2011end innovation engine\u2014spanning discovery, development, and in\u2011house GMP manufacturing\u2014creates a streamlined path to advance a diversified pipeline of one\u2011time 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<\/a>.<\/p> \nInvestors\/Business Development\n\n
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