Beam Therapeutics, Inc. announced the enrollment of the first patient in their BEACON clinical trial of BEAM-101, a genetic base editing treatment for individuals with sickle cell disease (SCD).

The trial, announced via news release, is an open-label, single arm, multicenter phase 1/2 trial to assess the safety and efficacy of the new therapy in adults with severe SCD.

“With the potency and precision of base editing, we believe BEAM-101 could be a best-in-class option for SCD patients with several advantages over other available genetic therapies,” wrote John Evans, chief executive officer of Beam. “We are now focused on activating additional clinical trial sites in the U.S., modifying the BEACON protocol to enable expedited future patient enrollment and endpoint assessment, and finalizing our commercial-ready manufacturing process.”

BEAM-101 is a novel autologous hematopoietic stem cell (HSC) treatment that is patient specific. It is designed to incorporate genetic nucleotide base edits that mimic the single-nucleotide polymorphisms observed in patients with genetic persistence of fetal hemoglobin. In preclinical studies, BEAM-101 achieved high levels of HSC editing and consistently upregulated fetal hemoglobin in addition to reducing levels of the pathogenic hemoglobin S protein.

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The advantages of BEAM-101 over nuclease editors include the fact that it offers precise and efficient single-base changes and avoids double-stranded DNA breaks in the editing process, which have been shown to lead to chromosomal abnormalities and genotoxic stress.

Initially, 3 patients will be enrolled as the “sentinel” cohort. Each patient in the BEACON trial will undergo HSC retrieval and pretreatment conditioning with chemotherapy. The HSC will be edited and then transplanted back into the patients. If successful engraftment is achieved, up to 45 patients in total will be dosed.

Reference

Beam therapeutics enrolls first patient in BEACON clinical trial of BEAM-101 base editing therapy candidate for the treatment of sickle cell disease. News release. Beam Therapeutics; November 14, 2022.