In vivo
HSC editing
IN VIVO HSC EDITING
Editing Approach:
Activation of HbF
Delivery:
In vivo LNP
Beam is exploring the potential for in vivo base editing programs for the treatment of sickle cell disease and beta-thalassemia. Using this approach, base editors would be delivered through intravenous infusion of lipid nanoparticles (LNP) targeted to hematopoietic stem cells, eliminating the need for transplantation.