Wiskott-Aldrich Syndrome (WAS) is an inherited immune deficiency disorder. It is characterized by hemorrhages, acute or chronic eczema, recurrent infections (airways, digestive tract, skin) due to common or opportunistic germs, and autoimmune manifestations.
Patients with WAS also have an increased risk of developing tumors at any age.
The incidence is estimated at less than 1/100,000 births. The disease almost exclusively affects boys and is life-threatening for those with severe forms.
For patients without a compatible donor, gene therapy based on correction of autologous HSC represents a major hope.
A team led by Dr Anne Galy (Genethon-INSERM Mixed Unit U951) has developed a lentiviral vector making it possible to obtain physiological levels of the WAS gene and has demonstrated the efficacy and safety of the vector in models of the disease.
Genethon is conducting an international clinical trial involving patients with severe forms of the disease; in France (Necker Children’s Hospital, Paris), in Great Britain (Great Ormond Street Hospital, London), and in the United States (Children’s Hospital Boston).
The trial, which began in 2011, has made it possible to treat 15 patients and in Europe, the last patient will complete the trial in Q3 2019. Following treatment, patients are followed for 10 years: 2 years in the main clinical trial and then 8 years in a tolerance monitoring trial.
Genethon obtained “Orphan Drug” status for this product from the European Medicines Agency (EMA) on 07/10/2013 under No. EU/3/13/1196.