RM001

RM001 is an investigational, autologous, ex vivo CRISPR-Cas9 gene-edited hematopoietic stem cell therapy developed by Reforgene Medicine for the treatment of transfusion-dependent β-thalassemia (TDT). The therapy involves the collection of a patient's CD34+ hematopoietic stem and progenitor cells (HSPCs), which are then edited using CRISPR-Cas9 to disrupt the BCL11A erythroid-specific enhancer binding site within the promoters of the γ-globin genes (HBG1 and HBG2). By preventing the binding of the transcriptional repressor BCL11A, RM001 reactivates the production of fetal hemoglobin (HbF). Following myeloablative conditioning, the edited cells are re-infused into the patient, where they engraft and produce red blood cells containing high levels of HbF, thereby reducing or eliminating the need for chronic blood transfusions.

Other names

Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells