Beam reports BEAM-101 Phase 1/2 data: all 7 patients achieved >60% HbF, resolved anemia; ESCAPE proof-of-concept in NHPs

Beam Therapeutics Inc.

Seven patients dosed with BEAM-101 achieved HbF >60%, HbS <40%, and resolution of anemia; no VOCs post-engraftment.
Mean neutrophil engraftment 17.1 days, platelet 19.1 days; one death from busulfan-related respiratory failure deemed unrelated.
ESCAPE antibody conditioning in NHPs enabled long-term engraftment of base-edited HSCs with >80% HbF-containing cells.
BEAM-103 mAb dosing was well tolerated without transfusions or supportive care; minor declines in neutrophils/platelets.
BEAM-302 initial clinical data from multiple cohorts now expected in first half of 2025.

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