Research Pre-clinical phase Phase I or I/II  

Genethon is sponsoring an international phase I/II clinical trial for Wiskott-Aldrich Syndrome (WAS), an inherited immune deficiency. Treatment is based on ex vivo gene transfer, whereby a lentiviral vector carrying the therapeutic gene is inserted into the patient’s hematopoietic stem cells.

This clinical study, initiated in February 2010 in London by the team of Professor Adrian Thrasher (Great Ormond Street Hospital), is also conducted in Paris by the team of Prof. Alain Fischer, Marina Cavazzana-Calvo and Salima Hacein-Bey (Hôpital Necker-Enfants Malades), and in Boston by the team of Professor David Williams (Children’s Hospital Boston).

Wiskott-Aldrich Syndrome presents as recurrent infections, hemorrhaging and eczema, and is life-threatening for children affected with severe forms of the disease. Gene therapy has already made it possible to treat other hereditary immune deficiencies and has created great hope for these young sufferers.

This clinical trial follows on from work that has been conducted at Genethon since 2002 by the multi-disciplinary team led by Dr Anne Galy (Genethon-INSERM Mixed Unit U951), and Genethon is in charge of producing the clinical-grade vector batches for the trial.

Genethon has obtained ‘Orphan Drug’ status for this product from the European Medicines Agency (EMA).


Press releases: