Beam Therapeutics announced on November 10 that ristoglogene autogetemcel, known as risto-cel and formerly BEAM-101, met the primary efficacy endpoint of the BEACON Phase 1/2 study in sickle cell disease: freedom from severe vaso-occlusive crises for at least 12 consecutive months, known as VF12, on independently adjudicated results. The study also met all primary safety endpoints at the prespecified interim analysis and all secondary endpoints, including freedom from inpatient hospitalisation for severe crises over 12 consecutive months. As of the October 20 data cutoff, 49 patients had been treated, 39 adults and 10 adolescents, and 31 had completed the follow-up required for the VF12 assessment.
Risto-cel is a one-time treatment made from a patient’s own blood stem cells, base-edited in the promoter regions of the HBG1/2 genes so that the repressor BCL11A cannot bind, which switches production of fetal hemoglobin back on. Unlike first-generation CRISPR treatments, base editing changes single DNA letters without cutting through both strands. Beam said safety findings were consistent with previously reported BEACON data and with the known effects of plerixafor mobilisation, busulfan conditioning and the transplant procedure itself. The company did not disclose what proportion of the 31 evaluable patients achieved VF12, nor detailed hemoglobin or adverse event figures. Detailed pivotal results are planned for a medical meeting in 2027, with data from an earlier cutoff to be presented orally at the American Society of Hematology meeting in New Orleans on December 12 to 15, 2026. In November 2024 Beam disclosed the death of a patient in BEACON from respiratory failure four months after treatment, a fact that remains part of the safety record the FDA will review.
Beam expects to submit a Biologics License Application to the FDA as early as the end of 2026, which it says has the potential to be the first filing for a base editing therapy. Approval would make risto-cel the third genetic therapy for sickle cell disease, after Vertex and CRISPR Therapeutics’ Casgevy and Genetix Biotherapeutics’ Lyfgenia, both of which sold slowly at first. BioPharma Dive reported that Vertex recorded 219 million dollars in Casgevy sales through the first nine months of 2026, after only 64 patients received treatment in 2025, and that Beam argues uptake has been limited by supply constraints rather than demand. Beam’s management case rests on a shorter manufacturing process and fewer mobilisation cycles. The market was not persuaded on the day: Beam shares fell as much as 10 percent in Tuesday morning trading, according to BioPharma Dive. Sickle cell disease affects an estimated 100,000 people in the United States and about eight million worldwide.