Genes, composed of DNA, serve as the “blueprint” for human structure and function. When genes malfunction, they may cause disease; such defective genes are typically referred to as mutated genes. Gene therapy is an innovative medical technology designed to treat or prevent disease by correcting gene mutations or dysfunctional gene activity, thereby restoring or improving normal physiological function. Today, gene therapy has demonstrated breakthrough benefits across multiple disease areas, including hemophilia, sickle cell disease, cancer, heart disease, and diabetes. 

GoBroad is currently focused on advancing clinical research and application of gene therapy in monogenic disorders such as transfusion-dependent β-thalassemia and sickle cell disease. These conditions are caused by abnormal hemoglobin production, resulting in severe chronic anemia. Gene therapy has become one of the world’s most successful and representative treatment breakthroughs for these diseases.

Both transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are caused by mutations in the β-globin gene, which result in abnormal hemoglobin structure or impaired hemoglobin synthesis. This leads to chronic anemia and multi-system complications.

Because the disease mechanism is clear and the defective gene is singular, gene therapy can correct the underlying genetic defect at the hematopoietic stem cell level, restoring normal red blood cell production. This makes it one of the most promising curative treatment strategies available today.

Treatment Process

1. Stem Cell Collection:

Hematopoietic stem cells (HSCs) are collected from the patient.

2. Gene Correction:

A lentiviral vector is used ex vivo to insert a functional β-globin gene into the patient’s HSCs.

3. Reinfusion & Reconstruction:

After conditioning, the corrected stem cells are reinfused. They engraft in the bone marrow and gradually restore normal hematopoiesis, enabling patients to become independent of chronic blood transfusions.

Lentiviral vector–based gene therapy typically requires only a single treatment, and many patients achieve long-term remission. Some remain transfusion-free for years, showing the potential for functional cure and dramatic improvements in quality of life and life expectancy.

• Thalassemia
• Sickle Cell Disease

GoBroad continues to lead the international forefront in the treatment of thalassemia and remains dedicated to providing patients with more advanced, safer, and more effective personalized therapeutic strategies. With a fully established hematopoietic stem cell transplantation platform, the transplantation team achieved a global milestone by applying TDH transplantation for severe thalassemia—becoming the first in the world to achieve a GVHD-free, thalassemia-free survival (GTFS) rate exceeding 80%, a result superior to all other published international data. With continuously optimized protocols, the team has achieved a 97.1% engraftment rate, an overall survival (OS) rate of 96.5%, and a thalassemia-free survival (TFS) rate of 94.3%.

Building on these exceptional transplantation outcomes, the team introduced internationally leading gene therapy products such as Hemo-cel, opening an entirely new curative pathway for patients who are not suitable candidates for transplantation or who continue to require treatment after transplantation.

In clinical studies, Hemo-cel has demonstrated strong efficacy and a favorable safety profile.

1. Faster engraftment and earlier recovery:

Among patients treated with Hemo-cel, the median neutrophil engraftment time was 18 days (range 16–22 days), and the median platelet engraftment time was 13 days (range 12–19 days). Compared with the internationally approved product ZYNTEGLO, the engraftment period was shortened by more than 50%, enabling patients to eliminate transfusion dependence more quickly, reduce hospitalization duration, and return to normal life sooner.

2. Durable efficacy with significant improvement:

Some patients have maintained a transfusion-free status for more than four years, and their iron overload has continued to improve even without the use of iron chelators. These findings indicate that GoBroad’s gene therapy not only corrects hemoglobin deficiencies but also provides long-term improvement in hematopoietic function and overall metabolism.

3. Safe and reliable with controllable quality:

Adverse reactions associated with Hemo-cel are consistent with those typically seen during myeloablative conditioning—such as thrombocytopenia, mucositis, fever, and anemia—and all can be fully resolved with supportive care. No cases of replication-competent lentivirus, clonal dominance, or insertional oncogenesis have been observed in any patient.

We uphold a “patient-first” philosophy and, through the combined strengths of transplantation technology and gene therapy, tailor scientific, systematic, and compassionate treatment plans for every patient—helping more families regain hope for a healthy future.