KL003

KL003 is an autologous hematopoietic stem cell gene therapy developed for the treatment of transfusion-dependent β-thalassemia and sickle cell disease. The therapy involves collecting a patient's own CD34+ hematopoietic stem cells, which are then transduced ex vivo with a lentiviral vector encoding the βA-T87Q-globin gene—a modified version of the human hemoglobin beta chain. After myeloablative conditioning, these genetically modified cells are reinfused into the patient to restore functional hemoglobin production and reduce or eliminate transfusion requirements. Clinical studies have shown that most patients treated with KL003 achieve transfusion independence with rapid engraftment and no serious adverse events reported so far. The product is currently in Phase 1/2 clinical trials in China and has received rare pediatric disease and orphan drug designations in the United States[1][3][5][7][8].

Other names

KL-003KL003KL 003βA-T87Q-globin gene therapy