Trial Profile

EXPEDITION: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 20 Jan 2026

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At a glance

Drugs ETX 101 (Primary)
Indications Dravet syndrome
Focus Adverse reactions; Therapeutic Use
Acronyms EXPEDITION
Sponsors Encoded Therapeutics

Most Recent Events

08 Jan 2026 According to Encoded Therapeutics media release, interim efficacy results from the highest dose level of the POLARIS clinical program expected in 2026.
12 Dec 2025 Planned number of patients changed from 4 to 5.
12 Dec 2025 Planned End Date changed from 1 Sep 2030 to 1 Oct 2030.

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