[China Tech] Gene-editing technology saves a young woman from Nigeria

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A Chinese gene-editing technology has helped cure a 21-year-old Nigerian student in Shanghai of congenital blood illness.

This is the first sickle cell disease patient in the country to benefit from the clinical trial of CS-101, a new high-precision base-editing medication that was introduced by Shanghai-based CorrectSequence Therapeutics Co Ltd and the First Affiliated Hospital of Guangxi Medical University.

To treat severe disorders like β-thalassemia and sickle cell disease, CS-101 employs revolutionary transformer Base Editing (tBE) technology.

Sickle cell disease, caused by β-globin gene abnormalities, causes sickle-shaped red blood cells, persistent anemia, pain, infection risk and organ damage. Approximately 3.5 percent of the global population has the sickle mutation, with 300,000 afflicted newborns born annually, primarily in Africa, the Mediterranean, the Middle East, and South Asia.

Blood transfusions and symptomatic medicines can relieve symptoms but not cure. Regenerative stem cell transplantation is possible, but matching donors are needed. Gene editing, especially base editing, can activate fetal hemoglobin in a patient's blood cells, eliminating the requirement for a donor and shortening and possibly improving treatment.

Following therapy with CS-101, the patient's fetal hemoglobin (HbF) levels significantly and persistently increased, whereas sickle hemoglobin (HbS) levels significantly decreased and stabilized at 35 percent six months later.

She became the first patient in China to successfully receive this novel therapy, and she was allowed to resume her studies and regular life after experiencing no vaso-occlusive crises (VOCs) during the six-month follow-up.