Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system

Genethon, which developed the lentiviral vector used and sponsored initial clinical studies, is pleased with the conclusive results of the gene therapy trial underway in the United States and England in X-linked Chronic Septic Granulomatosis (X-CGD), a rare and severe immune dysfunction. Six of the nine patients are free of treatments related to complications generated by the disease.

These results have been published in Nature Medicine ( Genethon, who had conducted initial preclinical and clinical studies had formed a strategic alliance with the British company Orchard Therapeutics which has an exclusive license on G1XCGD for the further clinical development of this gene therapy drug.

The lentiviral vector – G1XCGD – used was developed at Genethon by Dr. Anne Galy, in collaboration with Dr. Adrian Trasher of London and Prof. Manuel Grez of Frankfurt and the clinical batches were produced by YposKesi, the industrial production platform for gene therapy drugs created by AFM-Telethon and BPIfrance.

Within the framework of clinical trials conducted in the United Kingdom and the United States, nine patients (4 in Europe and 5 in the United States), aged between 2 and 27 years, were treated. Seven of them, followed for 12 to 36 months after treatment, did not contract any infection. Two people died during the trial as a result of complications acquired prior to gene therapy treatment. This international effort has also been supported by the European Commission through funding by the 7th Framework Programme in Health of the European project Net4CGD of which Genethon is the coordinator.

“We are very proud of these clinical results, which once again demonstrate the unique capacity of our laboratory to develop therapeutic projects, from concept development to clinical trials by integrating manufacturing. These results are also the result of a rich collaboration with the best British and American clinical experts.” said Frédéric Revah, Chief Executive Officer of Genethon.

“This is the first time that a sustainable treatment has been obtained by gene therapy in this disease, confirming the advantages of the lentiviral technology that has been used to treat hematopoietic stem cells,” says Anne Galy, Director of the Blood and Immune System Diseases Program at Genethon.

Find out more

Read the press release on the gene therapy trial in Chronic Septic Granulomatosis