Correctsequence Therapeutics Co., Ltd

This early-phase study tested a new gene-edited stem cell treatment (CS-101) in 5 people with beta-thalassemia, a severe blood disorder requiring regular blood transfusions. The goal was to see if the treatment is safe and can help patients produce healthy red blood cells, potent…

This early-stage study tested a single dose of a gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The main goals were to check safety and see if the treatment helps the body make more healthy hemoglobin. …