Hemo-cel gene therapy

Hemo-cel gene therapy is most promising to treat sickle cell disease and thalassemia because the gene has the mutation causing these diseases. With the introduction of a functional gene or through gene editing methods, Hemo-cel could correct faulty hemoglobin production, letting patients reduce or remove their symptoms of this disease entirely. Such therapy will offer tremendous improvement in the quality of life for such patients once and for all, contrasting with the perpetual renewal of the patient’s blood transfusions and medications.

What is gene therapy?

Gene therapy offers a potential cure for transfusion-dependent thalassemia (TDT) and severe sickle cell disease (SCD) through a single treatment. It involves taking hematopoietic stem and progenitor cells (HSPCs) from the patient and changing their genes so that they can make functional β-globin chains. This helps people with severe anemia and stops sickle red blood cells from clumping together. The process includes isolating CD34+ HSPCs, cultivating them, and transducing themwith a lentiviral vector. After a conditioning regimen depletes existing HSPCs, the modified cells are reinfused, allowing them to generate healthy hemoglobin and, consequently, produce functionally normal red blood cells. Gene therapy uses the patient’s own HSPCs, avoiding the need for HLA matching and reducing the risks of graft-versus-host disease (GVHD). Overall, this represents a significant advancement for TDT and SCD patients, offering hope for improved long-term health. Hemo-cel is a gene therapy designed for the treatment of TDT and SCD. It has received clinical evidence based on its demonstrated clinical efficacy and safety profile established during clinical trials conducted in China.

Why choose Hemo-cel?

Hemo-Cel

Hemo-cel is a gene therapy designed to treat transfusion-dependent thalassemia and sickle cell disease. It uses the patient’s own modified stem cells to produce healthy red blood cells that help balance hemoglobin levels. So far, Hemo-cel has cured five patients, with one enjoying nearly four years without needing blood transfusions. Approved for clinical trials in late 2023, Hemo-cel is also expanding its research internationally, including partnerships in Saudi Arabia, Thailand, and Malaysia.

Hemogen therapeutics

Hemogen, supported by the strong scientific research capabilities of BGI-Research and BGI Group’s channel influence in the medical and health science field, Hemogen is committed to the treatment of hemoglobin diseases and to “Create an Anemia-Free World Together”.

Hemo-cel Process

The mechanism of Hemo-cel

Hemo-cel consists of autologous CD34+ cells that have been genetically modified with hematopoietic stem and progenitor cells (HSPCs) transduced by a lentivirus named Lenti HBBT87Q . In general, the mechanism of action of Hemo-cel involves a lentivirus vector (LV) transferring the modified β-globingene into the genome of HSPCs from patients with β- thalassemia and sickle cell disease. Upon successful engraftment, HSPCs with the exogenous functional βA-T87Q-globin gene will differentiate into red blood cells expressing this modified globin. This expression helps restore the normal α/β globin ratio and hemoglobin function. Hemo-cel has the potential to produce functionally normal red blood cells, reducing the occurrence of anemia and preventing the aggregation of sickle red blood cells.

Treatment Process of Hemo-cel-Taking β-Thalassemia as an Example

Hemo-cel Clinical Data

Clinical Efficacy

Neutrophil engraftment (Days)

Platelet engraftment (Days)

Upon receiving Hemo-cel, the average time to engraftment for neutrophils was 19.4 days, and for platelets, it was 17.4 days. This represents a significant reduction of over 50% in the time required for HSCs engraftment when compared to Bluebird’s FDA approved product, Zynteglo. The accelerated engraftment enables patients to become independent of blood transfusions faster and markedly hastens the recovery of their hematopoietic system.

Last transfusion (Days)

Transfusion independence

The last blood transfusion was given about two weeks post-infusion, with no further transfusions required. Subjects P001 and P002 reached nearly 4 years of follow-up, maintaining transfusion independence. Other subjects also experienced significant periods without transfusions, and their iron overload status improved without chelation therapy, although ferritin levels remained elevated.

Clinical Safety Profile

The adverse events observed after Hemo-cel gene therapy infusion were consistent with those that are typical of busulfan-based myeloablation. The serious adverse reactions (>3%) included decreased platelet count, decreased blood cell count, decreased white blood cell count, decreased neutrophil count, oral mucositis, febrile neutropenia, anemia, and pharyngitis. These incidents were attributed to the conditioning regimen and were resolved with appropriate treatment. There were no detections of replication-competent lentivirus, clonal predominance, or insertional oncogenesis in any patient.

Development History

Since the initiation of research on the treatment of thalassemia in 2017, Hemogen Therapeutics was established in 2021. In 2022, the company began clinical studies in collaboration with hospitals conducting gene therapy for thalassemia. Following approval from the ethics committee at Shenzhen Children’s Hospital, two pediatric patients were treated successfully, leading to their complete independence from blood transfusions and clinical recovery. As of 2023, Hemogen has treated five beta-thalassemia patients in China, resulting in their complete cessation of transfusion dependence. By the end of 2023, Hemogen obtained China IND approval to commence clinical studies, with plans to complete Phase 1 clinical trials by the end of 2024. The company aims to be among the first in the country to achieve market approval, ultimately benefiting a larger patient population.

Global Clinical Application

History

Since its inception, Hemogen has consistently focused on the widespread application of thalassemia treatment. In 2023, the company established commercial partnerships in high-prevalence regions for thalassemia, including Southeast Asia (Thailand, Malaysia) and the Middle East (Saudi Arabia, UAE, Oman, Qatar), to provide treatment options for more patients.