autologous BCL11A-disrupted human hematopoietic stem and progenitor cells HIX763

A population of autologous human hematopoietic stem and progenitor cells (HSPCs) that are genetically modified to disrupt the activity of B-cell lymphoma/leukemia 11A (BCL11A), with potential usage for transplantation in patients with sickle cell disease (SCD). Upon infusion into the patient, the autologous BCL11A-disrupted HSPCs HIX763 can populate the bone marrow and differentiate into a variety of blood cell types including lymphoid cells, myeloid cells and erythroblasts. As BCL11A is a suppressor of fetal hemoglobin (HbF; hemoglobin F) expression, disruption of the expression of BCL11A stimulates the expression of HbF in erythrocytes that differentiate from HIX763. HbF may compensate for reduced or absent expression of adult hemoglobin (Hb) in patients with SCD. HbF is a form of the oxygen carrying Hb that is naturally present at birth and is then replaced by the adult form of hemoglobin.