The kick-off meeting of "HGI-001 Injection" in cooperation with Shenzhen University General Hospital was held successfully on November 15th, 2022.
Li Yu, chief expert of clinical medicine at Shenzhen University General Hospital, director of Shenzhen University Institute of Hematology, executive director of Shenzhen University International Cancer Center, director of Hematology Oncology Department; Lixin Wang, deputy director of Shenzhen Laboratory of Precise Diagnosis and Treatment of Malignant Hematology, deputy director of Hematology Oncology Department; Shuhong Wang, Deputy Chief Physician of the Transplant Ward; Dr. Chao Liu, CEO of Hemogen; Dr. Wenjie Ouyang, Head of R&D (Hemogen); Haigang Sun, Senior Project Manager (Hemogen); Dr. Nan Han, Medical Manager (Hemogen); Zhanxin Luo, Vice President of Guangdong Thalassemia Association Support Group; and other relevant personnel attended the meeting.
The treatment of β-thalassemia with gene therapy involves ex vivo gene transfer of functional β-globin to autologous hematopoietic stem cells (HSCs). HGI-001 injection is based on the idea that the HSCs are cultured ex vivo and then are subjected to gene transfer with an integrating lentiviral vector encoding functional β-globin. HGI-001 injection restores hemoglobin balance and thereby helps transfusion-dependent β-thalassemia (TDT) patients achieve transfusion independence (TI). HGI-001 injection is currently the top priority of Hemogen. The IITs (investigator-initiated trials) initiated in December 2020 has successfully helped four TDT subjects achieved TI. As of November 15th 2022, the subject who achieved TI for the longest period of time has been transfusion independent for 21 months.
Among the four subjects, the highest hemoglobin level reached 136g/L after gene therapy treatment. Current clinical data show that HGI-001 injection has a significant therapeutic effect for children with TDT. No serious adverse events (SAEs) related to HGI-001 injection have been found, suggesting that HGI-001 injection is safe and effective. The oldest subject was 15 years old when he/she received gene therapy treatment. Adult TDT patients have greater risks associated with allogeneic hematopoietic stem transplantation due to long-term blood transfusions and iron overload, and they have urgent needs to receive gene therapy. This clinical study launched at Shenzhen University General Hospital will recruit adult patients with TDT to verify the safety and effectiveness of HGI-001 injection for treating adults.
At the meeting, Dr. Chao Liu, Dr. Nan Han and Mr. Haigang Sun presented "Background Introduction of Thalassemia Gene Therapy and Clinical Summary of HGI-001 Injection", "Introduction of HGI-001 Injection Clinical Trials Program" and "GCP in Project Implementation" respectively. The participating experts actively discussed the implementation of the research plan which includes screening, mobilization, myeloablation, cell transportation, and human subject protections. The participating experts also discussed the problems found in the early stage of research in detail, laying a good foundation for the follow-up work.
This clinical trial is the first clinical study of thalassemia gene therapy for adult patients with TDT in China. The success of this study will expand the scope of thalassemia gene therapy and provide valuable clinical experience, laying a solid foundation for HGI-001 injection application in adult patients.
Hemogen is making every effort to promote HGI-001's investigational new drug application (IND), and will continue to cooperate with all parties to create a "thalassemia-free world".
