Beam Therapeutics Unveils New Safety, Efficacy Data From Sickle Cell Disease Candidate, Stock Gains

On Saturday, Beam Therapeutics Inc. (NASDAQ:BEAM) revealed new safety and efficacy data from its BEACON Phase 1/2 trial of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs).

The data were featured at the American Society of Hematology (ASH) Annual Meeting and Exposition.

Consistent with Beam’s previously announced data, updated data from seven patients treated with investigational base-editing therapy BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis.

No VOCs were reported post-engraftment.

Key highlights include the following:

• Rapid and Sustained Increases in Protective Fetal Hemoglobin (HbF): All patients achieved endogenous HbF levels exceeding 60% and reduction in corresponding sickle hemoglobin (HbS) below 40% that was durable. A pancellular distribution of HbF was observed after the elimination of transfused blood.
• Robust and Sustained Total Hemoglobin ...

Full story available on Benzinga.com