Beam Therapeutics (BEAM) announced the acceptance of multiple oral and poster presentations, including the first clinical data from a Beam program, at the 66th American Society of Hematology, ASH, Annual Meeting and Exposition, taking place December 7-10, 2024, in San Diego. Initial Results from the BEACON Clinical Study: Initial safety profile was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation; These data support base editing of the HBG1/2 promoters as a therapeutic modality for the treatment of SCD and the ongoing development of BEAM-101. Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial results demonstrated 98-99% of RBCs expressing HbF as early as Month 1, with near complete elimination of RBCs expressing solely HbS post-treatment with BEAM-101. The data showed reduction in RBC sickling, comparable to sickle cell trait, reduced cell adhesion and improved hemorheological properties of blood post-treatment with BEAM-101. CD117 Antibody Conditioning and Multiplex Base Editing: Data suggest that ESCAPE, combined with CD117 mAb conditioning and selection, can achieve long-term engraftment and induce high levels of HbF, offering a promising alternative to traditional genotoxic conditioning in autologous hematopoietic stem cell transplantation .
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