HGI-002

HGI-002 is an investigational gene therapy based on autologous hematopoietic stem cells genetically modified ex vivo with a lentiviral vector encoding functional alpha-globin genes. Developed by Shenzhen Hemogen, this therapy is designed for the treatment of transfusion-dependent alpha-thalassemia. The process involves collecting the patient’s own hematopoietic stem cells, modifying them outside the body to restore alpha-globin expression using a lentiviral vector (LentiHBA T>C), and reinfusing them into the patient. The goal is to correct the underlying genetic defect in alpha-thalassemia, restore balanced hemoglobin production, and reduce or eliminate dependence on blood transfusions[1][3][9]. Early clinical data indicate that at least one patient has achieved transfusion independence following treatment with HGI-002[7].

Other names

α-globin Restored Autologous Hematopoietic Stem CellsHGI-002 injectionHGI002 injectionHGI 002 injection