SHENZHEN Hemogen Therapeutic Co., Ltd.

α-Thalassemia is a genetic disease with high prevalence worldwide. About 5% of the world's population carries an α globin (HBA) gene variant. α-Thalassemia is caused by the deletion or mutation of HBA1 and HBA2 genes, affecting the development and function of red blood cells, resulting in varying degrees of anemia. If 3 α-globin genes are defective, the synthesis of α-globin is severely reduced, resulting in excess β-globin and forms abnormal globin tetramer β4 (called hemoglobin H, or HbH).

To relieve the symptoms of anemia, patients with severe HbH disease need to go through regular red-cell transfusion, which brings heavy financial burdens to patients and their families.

At present, there is no effective treatment for α-thalassemia except for allogeneic hematopoietic stem cell transplantation. Gene therapy has been proven to be safe and effective in a variety of diseases, namely transfusion-dependent β-thalassemia. This gives hope of gene therapy to cure α-thalassemia.

There is currently no established gene therapy for α-thalassemia. HGI has independently completed design and optimization of self-designed lentiviral vector.

In vitro experiments show that the lentiviral vector can effectively transduce hematopoietic stem cells, and induce α-globin expression under lineage differentiation conditions. HGI is actively promoting preclinical research. Safety and efficacy of this gene therapy are evaluated using an immunodeficient mouse model, which lays the foundation for the development of clinical trials.

TBA