BEAM-103 (Beam Therapeutics)

BEAM-103 (also known as ESCAPE) is an investigational base-editing cell therapy developed by Beam Therapeutics for the treatment of sickle cell disease and beta-thalassemia. The program utilizes a dual-action approach: it employs adenine base editing to target the HBG1/2 promoters to activate fetal hemoglobin (HbF) production, while simultaneously introducing a specific mutation in the CD117 (c-Kit) receptor. This CD117 modification is designed to make the edited hematopoietic stem cells (HSCs) resistant to a specific anti-CD117 antibody, allowing for a non-genotoxic conditioning regimen (the ESCAPE strategy: Engineered Stem Cell Antibody Paired Evasion). This approach aims to enable the selective depletion of diseased host HSCs while sparing the transplanted, edited cells, potentially reducing the toxicity associated with traditional busulfan-based conditioning. The program is being explored for both ex vivo and potential in vivo applications.