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Encoded Therapeutics to Start Dravet Syndrome Gene Therapy Trials in US, Australia

NEW YORK – Encoded Therapeutics has received regulatory approval to begin clinical trials of its experimental gene therapy for Dravet syndrome in the US and Australia, the biotech firm said Tuesday.

The South San Francisco, California-based company is developing ETX101, its lead gene therapy candidate, as a treatment for SCN1A-positive Dravet syndrome, a developmental disorder characterized by treatment-resistant seizures and neurodevelopmental stagnation, which is often caused by loss-of-function variants in the SCN1A gene.

ETX101 uses an adeno-associated virus serotype 9 vector to deliver a transgene that encodes an engineered transcription factor under the control of a cell-selective regulatory element that aims to increase expression of the endogenous SCN1A gene.

Encoded plans to begin a two-part Phase I/II dose-escalation trial called ENDEAVOR in the US for patients six months through 3 years of age with SCN1A-positive Dravet syndrome in the first half of this year. Concurrently, it aims to start a Phase I/II dose-escalation trial in Australia called WAYFINDER in patients ages 3 years to 7 years old. Investigators will monitor seizure burden and potential long-term improvements in neurodevelopment in both trials.

Encoded said it has aligned with the US Food and Drug Administration on plans for a two-part Phase I/II trial that could transition to a confirmatory study following the initial demonstration of safety and efficacy.