Thalassaemia is the world’s most common genetic blood disorder. China currently has 30 million thalassaemia gene carriers, while the global total reaches 350 million.
Patient with severe thalassaemia could confront a stark reality: regular blood transfusions, potential organ transplants, and even the risk of premature death.
If two carriers of the gene marry, their children have a 25 per cent chance of developing the disease.
The therapy used CS-101, a base-editing drug developed for thalassaemia patients by scientists at ShanghaiTech University using transformer Base Editor (tBE) technology.
Five Chinese patients with beta-thalassaemia who received CS-101 treatment rapidly restored haematopoietic function – the ability to produce healthy new blood cells. Total haemoglobin and fetal haemoglobin (HbF) levels rose quickly and stayed high, freeing them from blood transfusions.
