Encoded Therapeutics presented additional Phase I/II POLARIS data for ETX101, an AAV9-based gene regulation therapy intended to increase SCN1A expression in children with Dravet syndrome. Investigators reported a robust and dose-dependent antiseizure effect persisting through 52 weeks, including a median seizure reduction of about 76% at the highest dose level. The company also reported early signals of stronger responses in participants not receiving sirolimus, consistent with preclinical observations on protein expression damping. Beyond seizure outcomes, the update described potential improvements in developmental domains that are typically difficult to shift in Dravet syndrome, positioning ETX101 as a candidate aimed at altering both seizure burden and disease trajectory.
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