Correctsequence Therapeutics Co., Ltd

This early-stage study tested a single dose of CS-101 gene therapy in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The goal was to see if the treatment is safe and can help the body produce healthy hemoglobin. While promising, pat…

This early study tested a new gene therapy (CS-101) in 5 people with beta-thalassemia, a blood disorder requiring frequent transfusions. The treatment uses the patient's own stem cells, edited to produce healthy red blood cells. The main goals were safety and whether patients cou…