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ETX 101

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Drug Profile

ETX 101

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

Latest Information Update: 24 Feb 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

  • 13 Feb 2025 ETX 101 receives Fast Track designation for Dravet syndrome [Intraventricular,Infusion] (In children, In infants, Treatment-experienced) in USA
  • 07 May 2024 Pharmacodynamic data from a preclinical studies released by Encoded Therapeutics
  • 30 Apr 2024 Phase-I/II clinical trials in Dravet syndrome (In children, In infants, Treatment-experienced) in USA (Intraventricular) (NCT05419492)

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