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Wiskott-Aldrich syndrome gene therapy - Genethon

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Drug Profile

Wiskott-Aldrich syndrome gene therapy - Genethon

Alternative Names: WAS gene therapy - Genethon

Latest Information Update: 15 Feb 2018

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At a glance

  • Originator Genethon
  • Developer Childrens Hospital Boston; Genethon
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Wiskott-Aldrich syndrome
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Wiskott-Aldrich syndrome

Most Recent Events

  • 13 Nov 2019 Genethon completes a phase I/II trial for Wiskott-Aldrich syndrome in United Kingdom (IV) (NCT01347242) (ISRCTN46087965)
  • 28 Aug 2019 Wiskott-Aldrich syndrome gene therapy is still in phase I/II trial for Wiskott-Aldrich syndrome in France, USA, United Kingdom (IV) (NCT01347346) (NCT01410825)
  • 21 Sep 2015 Phase I/II development is ongoing in USA, France and United Kingdom

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