Beam reports initial BEAM-101 clinical data: engraftment, HbF >60%, one death from busulfan conditioning

Beam Therapeutics Inc.

BEAM-101 (sickle cell disease): 4 patients engrafted, median 17 days neutrophil, 20 days platelet; HbF >60% by month 1.
BEAM-101: One death from respiratory failure attributed to busulfan conditioning, not BEAM-101; DSMC and FDA reviewed.
BEAM-201 (T-ALL/T-LL): 2 of 3 patients achieved CRi/CR at doses <200 million cells; both eligible for stem cell transplant.
ESCAPE NHP data: CD117 mAb conditioning well tolerated; HbF reached ~85% F-cells by 35 weeks in both animals.
Initial data from BEACON trial also showed no VOCs post-treatment and normalization of hemolysis markers in all 4 evaluable patients.

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