Tackling Dravet Syndrome With Gene Therapy

“In the case of Dravet, parents of children with Dravet are not only caregivers, but also advocates and decision makers for their kids. So, it is imperative that we ask them what is important to them, because we want to design a clinical trial or clinical development program with them, not for them.”

Encoded Therapeutics is focused on finding treatments for Dravet syndrome (DS), starting with their lead program, ETX101. The disease-modifying gene therapy is designed to reduce seizures and extend survival by upregulating patients’ SCN1A gene, and therefore sodium channels within GABAergic inhibitory neurons.

While the first clinical trial of ETX101 is expected to begin at the end of 2021, the company is currently conducting an observational study of patients with DS titled ENVISION (NCT04537832). ENVISION’s goal is to further the understanding of the natural history and disease course of DS to be able to better develop therapies to treat it. The first of about 50 participants was enrolled in the study in April 2021 and the study will be completed in June 2024.

GeneTherapyLive spoke with Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, to learn more about the company's work in tackling DS from different angles and their focus on patient-focused drug development. He added that Encoded plans to announce initial data from the ENVISION study by the end of the year.

REFERENCE

Hood V. Natural history study of children with Dravet Syndrome begins. March 19, 2021. Accessed August 10, 2021. https://www.dravetfoundation.org/natural-history-study-of-children-with-dravet-syndrome-begins/