Genetically Modified Cell Therapy BEAM-101 Successful in Sickle Cell Disease Trial

Beam Therapeutics has announced new data from BEACON, its phase 1/2 clinical trial of BEAM-101, a genetically modified cell therapy, for the treatment of sickle cell disease (SCD).

Announced at the European Hematology Association (EHA) 2025 Congress, these data demonstrate the investigational base-editing therapy’s strong clinical profile. This was established previously in a presentation at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December of 2024, during which Beam Therapeutics presented a complete resolution of anemia in all 7 patients treated with BEAM-101. Additionally, all participants dosed in this earlier trial achieved the target cell dose with only 1-2 mobilization cycles, as well as experiencing rapid neutrophil and platelet engraftment.2

BEAM-101 is an investigational genetically modified cell therapy, consisting of autologous CD34+ hematopoietic stem and progenitor cells which have been base-edited in the promoter regions of the HBG1/2 genes. The 1-time treatment is administered via a hematopoietic stem cell transplant procedure and is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without interrupting BCL11A expression. This will lead to increased production of non- and anti-sickling fetal hemoglobin (HbF), mimicking the effects of naturally occurring variants in hereditary persistence of fetal hemoglobin.1

“These promising and consistent new data from the BEACON trial, now from 17 patients treated with BEAM-101, continue to reinforce the potential of this gene therapy to fulfill this unmet need,” said Ashish Gupta, MD, MPH, associate professor at the University of Minnesota, pediatric blood and marrow transplant physician, and an investigator in the BEACON trial. “Given this profile, BEAM-101 has the potential to further restore red blood cell health and function, reduce hospital time for patients, and decrease the overall burden of cell and gene therapy treatment for patients and providers.”1

Consistent with the data shown at ASH, all patients exhibited endogenous HbF levels exceeding 60% and a durable reduction in corresponding HbS below 40%. During follow-up, which ranged from .2 to 15.1 months, patients maintained a pancellular distribution of HbF-expressing cells, with HbF levels per cell above the sickling threshold. After elimination of transfused blood, patients also saw a rapid increase in total Hb levels and resolution of anemia. These changes were durable for up to 15 months.1

Additional results included normalization of key hemolysis markers, such as indirect bilirubin, lactate dehydrogenase, and haptoglobin, as well as reticulocytes. Erythropoietin levels also decreased to normal or near normal following treatment, which investigators noted as indicating substantial improvement in oxygen delivery to tissues. Exploratory biomarker assessments of red blood cell health demonstrated improvements compared to baseline across multiple parameters after treatment.1

The ongoing adult and adolescent cohorts of the BEACON trial have finished enrollment; 28 patients were dosed as of June 13, 2025, and Beam Therapeutics expects to dose 30 patients by mid-2025 and share additional data from the trial by the end of the year.1

“We remain highly encouraged by the potential of BEAM-101, with today’s data further building on its potential to deliver a transformative treatment for patients with SCD,” said John Evans, chief executive officer of Beam. “With enrollment now complete in both the adult and adolescent cohorts, we are focused on continuing to dose patients in this trial as we work toward our mission of delivering life-long cures.”1

References

1. Beam Therapeutics. Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Supporting Differentiated Profile in Sickle Cell Disease (SCD) at European Hematology Association (EHA) 2025 Congress. GlobeNewswire. June 13, 2025. Accessed June 23, 2025. https://www.globenewswire.com/news-release/2025/06/13/3098931/0/en/Beam-Therapeutics-Announces-New-Data-from-BEACON-Phase-1-2-Clinical-Trial-of-BEAM-101-Supporting-Differentiated-Profile-in-Sickle-Cell-Disease-SCD-at-European-Hematology-Associatio.html

2. Beam Therapeutics. Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 in Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting. December 7, 2024. Accessed June 23, 2025. https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-new-data-beacon-phase-12-clinical