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Angelman syndrome gene therapy - Encoded Therapeutics

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Drug Profile

Angelman syndrome gene therapy - Encoded Therapeutics

Latest Information Update: 13 May 2024

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At a glance

  • Originator Encoded Therapeutics
  • Class Gene therapies
  • Mechanism of Action UBE3A protein expression modulators
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Angelman syndrome

Most Recent Events

  • 23 Apr 2024 Preclinical trials in Angelman syndrome in USA (Intraventricular)

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