Encoded Therapeutics tied its ETX101 gene therapy for Dravet syndrome to a 78% reduction in seizures in Phase 1/2 data and signaled readiness to move into a pivotal study next year. The company presented the results as evidence of meaningful clinical benefit for a severe early‑onset epileptic encephalopathy with high unmet need. Encoded emphasized seizure reduction magnitude and durability in its briefing materials and outlined a regulatory path toward a registration study, subject to further manufacturing and long‑term safety data. Clarification: Dravet syndrome is a treatment‑resistant pediatric epilepsy often caused by SCN1A mutations; gene therapies aim to correct or compensate for the underlying genetic defect via targeted vector delivery to the CNS.
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