Wiskott-Aldrich syndrome gene therapy - Genethon

Drug Profile

Wiskott-Aldrich syndrome gene therapy - Genethon

Alternative Names: Ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS) - Genethon; Lentiviral vector containing the human Wiskott-Aldrich syndrome protein gene - Genethon; WAS gene therapy - Genethon

Latest Information Update: 21 Sep 2015

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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

  • 21 Sep 2015 Phase I/II development is ongoing in USA, France and United Kingdom
  • 21 Apr 2015 Interim efficacy data from a phase I/II trial in Wiskott-Aldrich syndrome released by Genethon
  • 01 Sep 2014 Genethon initiates a long term safety follow up trial for Wiskott-Aldrich Syndrome in France and United Kingdom (NCT02333760)
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