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BEACON Trial Achieved Primary Efficacy Endpoint of Freedom from Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months, Based on Independently Adjudicated Results, and Met All Primary Safety Endpoints at the Prespecified Interim Analysis
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Study Met All Secondary Endpoints Including Freedom from Inpatient Hospitalization for Severe VOCs for 12 Consecutive Months
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Results Support Planned U.S. Biologics License Application (BLA) Submission for risto-cel, Expected as Early as Year-End 2026
CAMBRIDGE, Mass., Nov. 10, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced positive topline results from the BEACON Phase 1/2 study evaluating ristoglogene autogetemcel (risto-cel) in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs). Risto-cel is an investigational one-time, autologous cell therapy with a potentially differentiated profile for the treatment of SCD.
BEACON achieved the primary efficacy endpoint of freedom from severe VOCs for at least 12 consecutive months (VF12) based on independently adjudicated results and met all primary safety endpoints at the prespecified interim analysis. As of the October 20, 2026, data cutoff, a total of 39 adults and 10 adolescents were treated in the BEACON trial, and 31 patients had completed the required follow-up period for the VF12 endpoint. The study also met all secondary efficacy endpoints, including freedom from inpatient hospitalization for severe VOCs for at least 12 consecutive months. The safety findings observed as of the data cutoff date were consistent with previously reported data from the BEACON study and with the known and intended effects of plerixafor use and the cell collection process, myeloablative conditioning with busulfan, the autologous hematopoietic stem cell transplantation (HSCT) process, and underlying SCD.
“These positive topline results represent a defining milestone for risto-cel and for Beam’s base editing technology more broadly, bringing us an important step closer to delivering a potentially transformative, one-time treatment for people living with sickle cell disease,” said John Evans, chief executive officer of Beam Therapeutics. “For patients with SCD, severe vaso-occlusive crises can be debilitating and life-threatening, making sustained freedom from these events an important clinical outcome. The achievement of our primary efficacy endpoint, supported by the broader clinical safety and efficacy profile observed in the BEACON trial, reinforces our confidence in risto-cel. With these results in hand, we are focused on submitting our planned BLA as early as year-end, which has the potential to be the first BLA for a base editing therapy.”
Beam expects to submit a Biologics License Application (BLA) for risto-cel to the U.S. Food and Drug Administration (FDA) as early as the end of 2026. Detailed pivotal results from the BEACON study are planned for presentation at a medical meeting in 2027.
Separately, Beam will present results from an earlier data cutoff from the BEACON clinical trial of risto-cel in SCD in an oral presentation at the 68th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 12–15, 2026, in New Orleans.
About ristoglogene autogetemcel (risto-cel, formerly known as BEAM-101)
Risto-cel is an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). The one-time therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promoter regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The risto-cel edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, which is intended to lead to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimic the effects of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. HbF is the predominant hemoglobin variant during development and early life. The safety and efficacy of risto-cel are being evaluated in the ongoing BEACON Phase 1/2 study, an open-label, single-arm, multicenter trial in patients with SCD with severe vaso-occlusive crises (VOCs).
About Sickle Cell Disease
Sickle cell disease (SCD), a severe inherited blood disease, is caused by a single point mutation, E6V, in the beta globin gene. This mutation causes the mutated form of sickle hemoglobin (HbS) to aggregate into long, rigid molecules that bend red blood cells into a sickle shape under conditions of low oxygen. Sickled cells obstruct blood vessels and die prematurely, ultimately resulting in anemia, severe pain (crises), infections, stroke, organ failure and early death. SCD is the most common inherited blood disorder in the United States (U.S.), affecting an estimated 100,000 individuals within the U.S. and approximately eight million people worldwide.
About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing lifelong cures to patients suffering from serious diseases.