Shenzhen Hemogen Therapeutics Co., Ltd. (hereinafter referred to as: HGI) cooperated with department of hematology of 923 Hospital on the topic of “Clinical Study on Safety and Effectiveness Evaluation of Autologous Stem Cell Injection Transduced by Lentivirus Encoding Human α-globin (HGI-002) in the Treatment of Transfusion-Dependent α-Thalassemia". On February 10, 2023, a kick-off meeting was held in 923 Hospital. Project PI, Director of Hematology Department Xinhua Zhang, Director of Hematology Department Xiaolin Yin, Chief Physician Yali Zhou, Director of Blood Transfusion Department Yaopeng Chen, Director of the Institutional Office Yuan Liang, Ethics Secretary of the Institutional Office Ling Ye, CEO of HGI Dr. Chao Liu, Senior clinical project manager of HGI Haigang Sun, and Medical manager of HGI Dr. Nan Han attended the kick-off meeting. 

HGI-002 Injection is a gene therapy based on autologous hematopoietic stem cells. By extracting patient's own hematopoietic stem cells and using lentivirus to reintroduce functional α-globin gene ex vivo, HGI-002 Injection helps to restore the ratio of α- and β-globin and to restore hemoglobin function in order to help patient achieve transfusion independence. HGI-002 Injection is another important research product of HGI, following the first-tier product HGI-001 injection (for the treatment of transfusion dependent β-thalassemia). 

This study will recruit patients with transfusion-dependent α-thalassemia to verify the safety and efficacy of HGI-002 injection. At the kick-off meeting, PI Zhang Xinhua and Director Yin Xiaolin introduced the background of α-thalassemia project, the situation of patients, and the general situation of the department. Dr. Liu Chao and Dr. Han Nan of HGI presented on the topics of "the preclinical test results of α-thalassemia gene therapy and the clinical progress of HGI-001 injection" and "the clinical plan of HGI-002 injection project". All participating experts actively conducted in-depth discussion on the curative effect and operational feasibility of splenectomy patients, bone marrow collection points, cell collections and backup stem cell storage, and stem cell transportation. The kick-off meeting has laid a solid foundation for the development of HGI-002 project.

At present, there is no case report of gene therapy for α-thalassemia worldwide. The launch of this research project is expected to help transfusion-dependent α-thalassemia patients achieve transfusion independence and to fill the gaps of α-thalassemia gene therapy. The success of HGI-001 Injection and HGI-002 Injection can effectively solve the plight of transfusion-dependent thalassemia patients who cannot be treated with allo-HSCT. The success of HGI-001 Injection and HGI-002 Injection also has the significance of achieving the goal of “a thalassemia-free world”.