NET4CGD Newsletter 2017 : Net4CGD Consortium - Gene Therapy for C-CGD
The Net4CGD European Consortium is focused on the clinical development of Gene Therapy as a new Orphan Drug for patients with the X-linked form of Chronic Granulomatous Disease (X-CGD). Patients receive autologous CD34+ blood stem cells transduced with the G1XCGD lentiviral vector.
In Europe, two clinical studies are ongoing: G1XCGD.01 study (NCT01855685) and G1XCGD.02 study (NCT02757911). Three patients have already been treated and results are encouraging.
Notably, a study is also ongoing in USA (NCT02234934) and three patient benefitted from the same therapeutic approach with good tolerance and clinical improvement.
For more information, please refer to the Net4CGD Newsletter 2017 that can be found here.