Chronic granulomatous disease (CGD) is a hereditary, recessive genetic disease (autosomic or X-linked). It is caused by a deficit in NADPH oxidase, an enzyme necessary for the function of phagocytes which are white blood cells responsible for the destruction of micro-organisms (bacteria, fungi…) entering the body.
CGD is characterized by the occurrence, early in life, of severe intractable and deep tissue infections that are resistant to standard therapy and lead to the formation of granulomas responsible for tissue complications.
Genethon is launching a project to treat CGD patients carrying mutations in the gp91Phox gene (3/4 of CGD patients). The objective is to restore NAPDH oxidase function in phagocytic cells following gene transfer into the patients’ own hematopoietic stem cells with a regulated lentiviral vector.
Genethon is sponsoring a multicentric international gene therapy trial in 4 countries to treat eligible CGD patients, in collaboration with renowned specialists of the disease and of gene therapy.
This clinical trial, sponsored by Genethon, will be conducted in parallel, in France at Hôpital Necker-Enfants Malades, in UK at the Great Ormond Street Hospital in London, in Germany at the University Hospital of Frankfurt and the Institute Georg-Speyer-Haus, and in Switzerland at the Children’s Hospital of Zurich.
The gene therapy vector is produced at Genethon.
Genethon has obtained “Orphan Drug” status for this product from the European Medicines Agency (EMA).