ET-01

ET-01 is an investigational autologous cell therapy consisting of CD34+ hematopoietic stem cells that have been edited using CRISPR/Cas9 technology. The editing process is designed to disrupt the BCL11A erythroid enhancer, which normally suppresses the production of fetal hemoglobin (HbF) in adults. By disrupting this enhancer, ET-01 reactivates the expression of γ-globin, leading to the production of HbF, which can compensate for the defective β-globin in patients with β-thalassemia. The therapy is being developed by EdiGene in collaboration with the Institute of Hematology & Blood Diseases Hospital in China specifically for the treatment of transfusion-dependent β-thalassemia (TDT). Preliminary clinical data from Phase 1 trials have demonstrated timely engraftment and the achievement of transfusion independence in treated patients.

Other names

BCL11A Enhancer modified Autologous Hematopoietic Stem CellsBCL-11A Enhancer modified Autologous Hematopoietic Stem CellsBCL 11A Enhancer modified Autologous Hematopoietic Stem Cells