A First in China: Shanghai's Independently Developed Gene Editing Technology Cures Foreign Patient

China's first foreign patient with β-thalassemia to undergo base editing treatment (Zhengxu Bio's CS-101) has been free from transfusion dependency for over two months and has returned to a normal life.

China's first foreign patient with β-thalassemia to undergo base editing treatment (Zhengxu Bio's CS-101) has been free from transfusion dependency for over two months and has returned to a normal life.

Today, Zhengxu Bio (Shanghai) announced that its independently developed gene editing drug, CS-101, has successfully cured a severe foreign β-thalassemia patient, marking China's first gene editing cure for a foreign patient. The patient has been free from transfusion dependency for over two months, with a stable total hemoglobin concentration above 120 g/L, and has returned to normal life.

To date, several β-thalassemia patients treated with CS-101 have successfully overcome transfusion dependency, with the longest case exceeding eight months. The 18-year-old Laotian patient, who received CS-101 treatment, previously required two units of red blood cells per month due to transfusion dependency. Now, this patient has achieved the goal of being free from transfusion dependency.

Zhengxu Bio (Shanghai) is an innovative biopharmaceutical company incubated by ShanghaiTech University, specializing in new gene editing technologies. In April of this year, Zhengxu Bio's CS-101 injection received approval from the National Medical Products Administration (NMPA) to officially begin clinical trials.

The principle behind the CS-101 therapy involves collecting the patient's own hematopoietic stem cells, using a high-precision base editor (BE) developed by ShanghaiTech University to precisely edit the patient's hematopoietic stem cells. This editing simulates naturally occurring beneficial base mutations found in healthy individuals, restoring the oxygen-carrying function of hemoglobin. After completing base correction, these hematopoietic stem cells are reinfused into the patient's body, where they produce healthy hemoglobin, allowing the patient's hemoglobin concentration to reach the level of a healthy individual and eliminating the need for transfusions.

Compared to traditional transfusion therapy and allogeneic hematopoietic stem cell transplantation, CS-101 injection is derived from the patient's own stem cells, with a shorter preparation time and no need for long-term waiting. Compared to other β-thalassemia gene editing therapies based on CRISPR technology, CS-101 injection offers better efficacy and safety, and more efficiently activates fetal hemoglobin expression, enabling patients to rapidly complete hematopoietic reconstruction and more quickly achieve transfusion independence.

Hemoglobinopathies are the most common single-gene hereditary diseases globally, with β-thalassemia and sickle cell anemia being the most prevalent. Approximately 7% of the world's population carries abnormal hemoglobin genes, with around 400,000 babies born each year with hemoglobinopathies. In China, there are about 30 million carriers of thalassemia gene mutations, with approximately 300,000 patients suffering from severe and intermediate forms of the disease, mostly located in the southern regions.

It is reported that clinical trials of CS-101 injection for treating sickle cell anemia are in preparation, and a global recruitment plan for patients with sickle cell anemia has already been launched.