GLOBE

GLOBE is an autologous ex vivo gene therapy developed for the treatment of transfusion-dependent beta-thalassemia (TDT). The therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that are harvested from the patient and transduced with the GLOBE lentiviral vector. This vector encodes the human beta-globin (HBB) gene under the control of the human beta-globin promoter and intragenic enhancers. Once the modified cells are re-infused into the patient following myeloablative conditioning, they engraft in the bone marrow and differentiate into red blood cells that produce functional adult hemoglobin (HbA). This process is designed to correct the hemoglobin deficiency and eliminate or significantly reduce the requirement for chronic blood transfusions. The program was originally developed by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and was previously licensed to Orchard Therapeutics before rights were returned to Fondazione Telethon, which is now pursuing a pioneering non-profit model for its commercialization.

Other names

GLOBE lentiviral vectorGLOBE-transduced CD34+ cells