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.
- The company also presented data in non-human primates at ASH for its next-generation candidate administered with a proprietary conditioning agent, which could eliminate the need for conditioning with busulfan
Respiratory adverse events, such as the idiopathic pneumonia syndrome that resulted in the death of the BEACON trial participant, are among the known risks of busulfan treatment, which SCD patients participating in Beam’s trial were warned about prior to enrolling in the study. CEO John Evans told Scrip that the company informed BEACON participants and prospective participants about
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