Correctsequence Therapeutics Co., Ltd

This early-stage study tested a new one-time treatment for beta-thalassemia, a serious inherited blood disorder. Researchers collected a small number of patients' own blood stem cells, used gene editing to 'switch on' a protective fetal hemoglobin gene, and then returned the cell…

This early-stage study tested a new, one-time gene therapy called CS-101 in 9 patients with a severe inherited blood disorder called beta-thalassemia major. The goal was to see if the treatment was safe and if it could help the patients' bodies make healthy red blood cells on the…