Correctsequence Therapeutics Co., Ltd

This early-stage study tested a new one-time gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe inherited blood disease. Doctors collected each patient's own blood stem cells, used gene editing to fix the underlying genetic cause, and then gave them back…

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…