Wiskott-Aldrich syndrome

Wiskott-Aldrich syndrome is a hereditary disease of the immune system. It is characterized by hemorrhages, recurrent infections and eczema. It can be a life-threatening disease in children who have the severe form of the disease.

International phase III clinical trial by ex-vivo gene transfer

  • Reference laboratory: Genethon
  • Partners: Great Ormond Street Hospital in London, Hopital Necker-Enfants Malades in Paris, Boston Children’s Hospital

In 2011 Genethon commenced an international Phase I/II clinical trial for Wiskott-Aldrich syndrome (WAS), a hereditary immune deficit. The treatment is based on ex vivo gene transfer utilizing a lentiviral vector introduced into hematopoietic stem cells of the patient. This trial is taking place simultaneously in London, Paris and Boston. It is the result of research carried out at Genethon since 2002 by a multi-disciplinary group directed by Dr. Anne Galy (Mixed Research Group INSERM-Genethon U951). The production of clinical vector batches in the trial was done at Genethon.

For more information: International PhaseI/II clinical trial by ex vivo gene transfer