Shanghai-based biotech company, CorrectSequence Therapeutics, in collaboration with the First Affiliated Hospital of Guangxi Medical University, has achieved a significant milestone in the field of medical science. Their clinical research on the alkaline editing drug CS-101, which targets severe beta-thalassemia, has resulted in the successful cure of a foreign patient, marking a remarkable achievement in China’s gene editing therapy sector.

Prior to receiving CS-101 treatment, the 18-year-old patient from Laos required blood transfusions at a rate of 2 units of red blood cells per month (equivalent to the amount of red blood cells extracted from 400 milliliters of blood). The patient has been free from transfusion dependence for over two months now, which marks the first instance of gene editing curing of a foreign patient in China.

To date, several beta-thalassemia patients have undergone the proprietary alkaline editing treatment developed by the Shanghai company, achieving independence from blood transfusions for extended periods, with the longest case exceeding 8 months.

The therapy involves the use of a precise deformed alkaline editing that was developed independently by ShanghaiTech University to edit the patient's autologous hematopoietic stem cells accurately in order to rebuild the oxygen-carrying function of hemoglobin. The edited hematopoietic stem cells are then reinfused into the patient's body, enabling the patient's hemoglobin concentration to reach levels comparable to those of healthy individuals, thus freeing them from transfusion dependence.

The therapy was approved by the National Medical Products Administration and initiated Phase I clinical trials in April.

Compared to traditional blood transfusion therapy and allogeneic hematopoietic stem cell transplantation, the CS-101 injection utilizes the patient's autologous stem cells, with a short preparation cycle, thus eliminating prolonged patient waiting times, according to CorrectSequence Therapeutics.

Hemoglobin disorders are among the most prevalent single-gene inherited diseases worldwide, with beta-thalassemia being a common hereditary hemoglobin disorder in southern China, coastal Mediterranean countries, South Asia, and Southeast Asia. It is estimated that approximately 30 million people in China carry beta-thalassemia mutation genes. Another common inherited hemoglobin disorder is sickle cell anemia, which is widely prevalent around the world, with approximately 300,000 infants born with sickle cell anemia each year.

Clinical trials of the CS-101 injection for sickle cell anemia treatment are currently in the preparatory stages, with a global recruitment program for sickle cell anemia patients already underway, according to CorrectSequence Therapeutics.

Source: shobserver.com