On December 19, 2023, Hemogen Biotechnology Co., Ltd. (Hemogen), based in Shenzhen, successfully obtained the IND（investigational new drug) approval from the National Medical Products Administration (NMPA) of China for its independently developed HGI-001 injection. The indication for this product is transfusion-dependent β-thalassemia (Acceptance No.: CXSL2300659).
This product utilizes a self-designed lentiviral vector to transduce patient hematopoietic stem cells ex vivo, allowing the modified hematopoietic stem cells to efficiently express β-globin during the process of erythroid differentiation. This addresses the functional defect caused by the genetic mutation, achieving functional cure for β-thalassemia.
The HGI-001 injection is a gene therapy product based on autologous hematopoietic stem cells. Through ex vivo genetic modification of the patient's defective hematopoietic stem cells using a lentiviral vector, the HBB gene expression element carried by the lentiviral vector can stably integrate into the genome of hematopoietic stem cells. This element contains red cell-specific promoters and transcriptional regulatory sequences, ensuring maximum levels and specificity of β-globin expression. Upon transplantation of the modified hematopoietic stem cells back into the patient, the newly differentiated red blood cells will be able to restore the proportion of β-globin and the function of hemoglobin, thereby reducing the patient's dependence on transfusions and achieving the goal of treating β-thalassemia.
Hemogen's HGI-001 has achieved significant results in Investigational Investigational Trials (IITs), with five treated patients successfully freed from transfusions. Without external transfusions, the hemoglobin levels of these patients remained above 90g/L for at least 12 months. The median time of transfusion independence for the five subjects was 34 months, 30 months, 19 months, 16 months, and 12 months, respectively. The median hemoglobin at the last visit for these five patients was 105 (range: 95~142g/L). The patient’s iron overload condition has also been alleviated, and no serious adverse events have occurred. This indicates that the HGI-001 injection has a good therapeutic effect and potential, providing a new treatment option for patients. The approval of the IND for HGI-001 injection will undoubtedly accelerate the product's development and benefit more thalassemia patients.
Hemogen currently has multiple product pipelines under research based on two major technology platforms (gene delivery and gene editing), involving α-thalassemia, immunodeficiency, and neurodegenerative diseases. Among them, the HGI-002 injection for α-thalassemia has entered the IIT clinical trial stage and successfully led one patient to transfusion independence. The company will continue to focus on clinical value, accelerate the research and development progress of pipeline products, and expedite the entry of more high-quality gene therapy products into the clinical stage.
Adhering to BGI's mission of "genetic technology benefits to humanity," Hemogen is committed to achieving the goal of "No Thalassemia in the World" and providing accessible and affordable gene therapy products for global hemoglobin disease patients. With the gradual progress of clinical trials, Hemogen will focus on expanding into the international market (already signed cooperation agreements with countries/regions such as Malaysia, Thailand, and Saudi Arabia). The company will seek to establish strategic partnerships with international collaborators to promote the development and commercialization of HGI-001 globally. Through multinational cooperation, Hemogen plans to introduce its innovative products into more countries and regions in the future, providing more treatment options for patients worldwide.
