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Summary:Returned to normal life

On July 22, a reporter from Jiefang Daily and Shangguan News learned from Zhengxun Biotechnology (Shanghai) that the clinical research on the base editing drug CS-101 for severe β-thalassemia they conducted in cooperation with the First Affiliated Hospital of Guangxi Medical University successfully cured the first foreign patient, who was free from blood transfusion dependence for more than two months. The total hemoglobin concentration was stabilized at more than 120 grams/liter and has returned to normal life, marking the first time in China that a foreign patient has been cured by gene editing.

So far, several β-thalassemia patients have been freed from blood transfusion dependence after base editing treatment with the proprietary intellectual property rights of Zhengxu Biotechnology, and the longest has lasted for more than 8 months. Before receiving CS-101 treatment, an 18-year-old Laotian patient had a blood transfusion frequency of 2 units of red blood cells per month (equivalent to the number of red blood cells extracted from 400 ml of blood), and has now achieved the goal of being free from blood transfusion dependence.

In April this year, Zhengxu Bio's CS-101 injection was approved by the National Medical Products Administration and started Phase I clinical trials. The principle is to use the high-precision variant base editor independently developed by Shanghai University of Science and Technology to perform precise base editing on the patient's autologous hematopoietic stem cells, simulate the beneficial base mutations naturally present in healthy people, and rebuild the oxygen-carrying function of hemoglobin. The edited hematopoietic stem cells are then infused back into the patient's body, allowing the patient's own hemoglobin concentration to reach the level of healthy people, thereby getting rid of dependence on blood transfusions.

Compared with traditional blood transfusion therapy and allogeneic hematopoietic stem cell transplantation, CS-101 injection comes from the patient's autologous stem cells, has a short preparation cycle, and patients do not need to wait for a long time.

Compared with other gene editing therapies based on CRISPR technology, CS-101 injection has better effectiveness and safety, will not cause double-strand breaks in DNA, and effectively avoids the risks of large DNA fragment deletions, chromosomal translocations, off-target mutations, etc.

Hemoglobinopathy is the most common single gene genetic disease in the world. Among them, β-thalassemia is a hereditary hemoglobinopathy prevalent in southern my country. It is also common in countries along the Mediterranean coast, South Asia and Southeast Asia. According to statistics, there are about 30 million carriers of the thalassemia mutation gene in my country; another common hereditary hemoglobinopathy is sickle cell anemia, which is widely present in many countries around the world. Every year, about 300,000 babies are born with sickle cell anemia.

It is reported that clinical trials on CS-101 injection for the treatment of sickle cell anemia are also being prepared, and a global recruitment plan for patients with sickle cell anemia has been launched.