Correctsequence Therapeutics Co., Ltd

This early-stage study is testing a potential one-time treatment for severe sickle cell disease. Doctors will collect a patient's own blood stem cells, edit a specific gene in the lab to boost protective fetal hemoglobin, and then return the cells. The goal is to see if this sing…

This is a very early safety study for a new, one-time gene-editing treatment called CS-121. It aims to help adults with a rare, inherited condition where the body cannot properly clear fat from the blood, leading to dangerously high levels and painful, life-threatening pancreas a…