ASH: Beam’s Base Editor Efficacy In Sickle Cell Disease Holds Up

Next-Gen Program Aims To ESCAPE Busulfan Conditioning

Beam presented data from three more patients than during its last update for BEAM-101, showing stem cell engraftment and efficacy consistent with the first four patients.

Base editing may give Beam a competitive edge in sickle cell disease (Shutterstock)

Beam Therapeutics reported promising efficacy in November for the first four sickle cell disease (SCD) patients treated with its base edited cell therapy BEAM-101 in the ongoing Phase I/II BEACON clinical trial, despite one death associated with busulfan conditioning used to prepare patients for treatment. In data reported on 8 December at the American Society of Hematology (ASH) annual meeting in San Diego, efficacy seen in three more patients was consistent with the first four, without another major busulfan-related event.

Key Takeaways

Beam Therapeutics’ base-edited cell therapy BEAM-101 showed consistent efficacy in additional sickle cell patients treated in the Phase I/II BEACON trial relative to a prior update.

Respiratory adverse events, such as the idiopathic pneumonia syndrome that resulted in the death of the BEACON trial participant, are...