Beam reports positive pivotal data for base-edited sickle cell therapy

Beam Therapeutics reported positive topline results from a pivotal phase 1/2 trial of ristoglogene autogetemcel (risto-cel), an investigational, one-time, autologous base-edited cell therapy for sickle cell disease (SCD).

The BEACON trial met its primary efficacy endpoint of freedom from severe vaso-occlusive crises (VOCs) for at least 12 consecutive months, based on independently adjudicated results, as well as all primary safety endpoints at a prespecified interim analysis.

As of the October 20, 2026 data cutoff, 39 adults and 10 adolescents had received risto-cel, and 31 patients had completed the required follow-up for the primary endpoint. The study also met all secondary efficacy endpoints, including freedom from inpatient hospitalization for severe VOCs for at least 12 consecutive months.

Beam expects to submit a BLA for risto-cel to the FDA as early as the end of 2026

Risto-cel (formerly BEAM-101) is designed to increase production of fetal hemoglobin (HbF), which can prevent red blood cells from sickling. The therapy consists of a patient's own CD34+ hematopoietic stem and progenitor cells, which are base-edited in the promoter regions of the HBG1 and HBG2 genes. The edits are intended to prevent the transcriptional repressor BCL11A from binding to the promoter regions, increasing HbF production without disrupting BCL11A expression.

Safety findings were consistent with previously reported BEACON data and the known effects of the treatment process, including plerixafor mobilization and cell collection, busulfan myeloablative conditioning and autologous hematopoietic stem cell transplantation.