CS-101

CS-101 is an autologous CD34+ hematopoietic stem cell therapy developed using in vitro base editing technology. The therapy specifically edits the BCL11A binding site in the promoter region of the HBG (hemoglobin subunit gamma) gene, preventing BCL11A from binding and thereby reactivating γ-globin (fetal hemoglobin, HbF) production. This increase in HbF compensates for deficient adult hemoglobin (HbA), aiming to achieve a clinical cure for β-thalassemia and sickle cell disease. CS-101 uses transformer Base Editor (tBE) technology, which allows precise DNA modification with reduced risk of large deletions or off-target effects compared to other CRISPR-based approaches. The product is manufactured from a patient’s own cells and addresses challenges such as donor matching and graft-versus-host disease associated with allogeneic transplantation[1][4][5][7].

Other names

CS-101CS101CS 101