Correctsequence Therapeutics Co., Ltd

This early-stage study tests a single injection of CS-101, made from a patient's own gene-edited blood stem cells, for severe sickle cell disease. The goal is to see if it is safe and can reduce or eliminate painful vaso-occlusive crises. The study involves 5 participants aged 12…

This early-stage study tests a new treatment called CS-121 for people with familial chylomicronemia syndrome (FCS), a rare genetic condition causing extremely high blood fats and risk of pancreatitis. CS-121 uses gene-editing technology delivered via lipid nanoparticles to target…