BEAM-101

BEAM-101 is an investigational, patient-specific, autologous hematopoietic stem cell therapy developed by Beam Therapeutics for the treatment of sickle cell disease (SCD) and potentially beta-thalassemia. The therapy uses ex vivo base editing technology to introduce precise A-to-G substitutions in the promoters of the HBG1/2 genes within CD34+ hematopoietic stem cells. This disrupts binding of the transcriptional repressor BCL11A without affecting its expression, leading to increased production of fetal hemoglobin (HbF). Elevated HbF levels can compensate for defective adult hemoglobin and reduce or eliminate sickling and related complications in SCD. The edited cells are transplanted back into the patient as a one-time intravenous infusion following myeloablative conditioning[1][2][3][4][5][6][8].

Brand names

Risto-cel

Other names

ristoglogene autogetemcelristo-cel