Correctsequence Therapeutics Co., Ltd

This early-stage study tests a new gene therapy (CS-101) for people with beta-thalassemia, a severe blood disorder requiring frequent transfusions. The treatment uses the patient's own blood stem cells, edited in a lab to produce healthy hemoglobin. The goal is to see if it is sa…

This study checks the long-term safety and effectiveness of a treatment called CS-101 in 8 people with beta-thalassemia who already received it in an earlier study. Researchers will monitor serious side effects and see if patients can stay free from blood transfusions for at leas…

This study follows 5 people with beta-thalassemia who already received CS-101, a gene therapy that modifies their own blood stem cells. Researchers will monitor them for years to check for side effects and see if they remain free from needing blood transfusions. No new treatment …