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Angelman syndrome gene therapies - Asklepios BioPharmaceutical/University of North Carolina at Chapel Hill

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Drug Profile

Angelman syndrome gene therapies - Asklepios BioPharmaceutical/University of North Carolina at Chapel Hill

Alternative Names: AAV-based Angelman syndrome therapies - Asklepios BioPharmaceutical/University of North Carolina at Chapel Hill

Latest Information Update: 28 Apr 2024

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At a glance

  • Originator University of North Carolina at Chapel Hill
  • Class Gene therapies
  • Mechanism of Action Gene transference; UBE3A protein expression stimulants
  • 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

  • No development reported Angelman syndrome

Most Recent Events

  • 28 Apr 2024 No recent reports of development identified for preclinical development in Angelman-syndrome in USA (Parenteral)
  • 18 Mar 2020 Preclinical trials in Angelman syndrome in USA (Parenteral), before March 2020

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