Drug Profile

ETX 101

Alternative Names: AAV-ETX; Dravet syndrome viral gene therapy - Encoded Therapeutics; ETX101

Latest Information Update: 05 Sep 2025

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At a glance

Originator Encoded Therapeutics
Class Antiepileptic drugs; Gene therapies
Mechanism of Action Gene transference; SCN1A protein expression stimulants

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Dravet syndrome
New Molecular Entity No

Highest Development Phases

Phase I/II Dravet syndrome

Most Recent Events

02 Sep 2025 ETX 101 receives Regenerative Medicine Advanced Therapy (RMAT) status for Dravet syndrome in USA
02 Sep 2025 Encoded Therapeutics plans pivotal clinical trials for Dravet syndrome, in 1H 2026
13 Feb 2025 ETX 101 receives Fast Track designation for Dravet syndrome [Intraventricular,Infusion] (In children, In infants, Treatment-experienced) in USA

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