Sickle cell patient dies in gene-therapy trial

Beam Therapeutics, based in Cambridge, Mass., released early clinical data from its first trial for patients with sickle cell disease that employed a gene editing tool, known as CRISPR. All four patients showed positive results, but one died four months post-treatment, according to a Nov. 5 news release from the company.

Data from the phase 1/2 Beacon trial indicated that Beam-101, the company’s sickle cell therapy, increased fetal hemoglobin levels from an initial range of 4% to 17% to levels of 72% to 74% within two months. Hemoglobin levels remained elevated for two patients at five- and six-month follow-ups.

One patient died four months after the Beam-101 infusion due to respiratory failure. The death was likely related to busulfan conditioning and was determined to be unrelated to the treatment itself.

No other grade 3 or higher adverse events or serious adverse events related to the treatment were reported.

Beam Therapeutics plans to expand the trial to a cohort of 42 participants.

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