BEAM-101 / Beam Therap - LARVOL DELTA

Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (BEAM-101) in the ongoing BEACON study (ASH 2025) - P1/2 | "A highernumber of sickle cell crises was observed during the mobilization period in WB (3 AEs in 2 pts) vs FD (1 AEin 1 pt).ConclusionIn the BEACON study, pts receiving plerixafor using an FD regimen required fewer days and cycles ofapheresis than pts on the traditional WB dose regimen. More CD34+ cells were apheresed in pts receivingFD due to the greater number of CD34+ cells mobilized in the peripheral blood, particularly on Day 1.Pre-dose CD34+ levels were similar, with a generally comparable AE profile between WB and FD groups."

Clinical • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34 • CXCR4

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study (ASH 2025) - P1/2 | "After mobilization with weight-based or tiered fixed-dose plerixafor, autologous CD34+ HSPCswere collected by leukapheresis and genetically modified with an adenine base editor. Additionally, there is a correspondingreduction in HbS levels to <40%, and improvements or resolution of hemolysis parameters and anemia,along with resolution of investigator-reported sVOCs post-engraftment. The safety profile of the BEAM-101 treatment regimen is consistent with busulfan conditioning, autologous HSCT, and underlying SCD.These results demonstrate the potential for base editing of the HBG1/2 promoters as a safe and effectivetherapeutic modality for the treatment of SCD that also has the potential to minimize hospitalization andtreatment process burden for patients, families, and sites."

Clinical • Anemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Neutropenia • Respiratory Diseases • Sickle Cell Disease • CD34

Sarah Cannon Research Institute to Present Research on Advances in…Blood Disorders at 2025 ASH Annual Meeting & Exposition (Sarah Cannon Research Institute (SCRI))

Clinical data • Beta-Thalassemia • Chronic Graft versus Host Disease • Sickle Cell Disease

Highlights of JTCC Research to be presented at ASH 2025: Noncancerous Blood Disorders (PRNewswire) - "End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children (ABSTRACT 25-4324); Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study (ABSTRACT 25-2531)."

Clinical data • Amyloidosis • Sickle Cell Disease • Thrombocytopenia

Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial Data from the BEACON Phase 1/2 Study of Autologous CD34+ Base Edited Hematopoietic Stem Cells in Sickle Cell Disease (ASH 2024) - "Finally, improvement in RBC cell number and resolution of abnormal RBC morphology were observed post-BEAM-101. In summary, the initial RBC functional biomarker data support base editing of the HBG1/2 promoters as a potentially transformative therapeutic modality for the treatment of SCD and will continue to be investigated in the ongoing BEACON study."

P1/2 data • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34 • HBG1 • VCAM1

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises (ASH 2024) - P1/2 | "After plerixafor mobilization, autologous CD34+ HSPCs were collected by leukapheresis/isolation and genetically modified with an adenine base editor...One patient died due to respiratory failure, likely related to busulfan conditioning, 4 months after infusion...These initial data support base editing of the HBG1/2 promoters as an effective therapeutic modality for the treatment of SCD and will continue to be investigated in the ongoing BEACON study. Updated data to be presented."

Clinical • P1/2 data • Anemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Respiratory Diseases • Sickle Cell Disease • CD34 • HP

Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease (GlobeNewswire) - "...the company plans to present updated data from the BEACON Phase 1/2 trial by the end of 2025."

FDA event • P1/2 data • Sickle Cell Disease

BASE EDITING FOR SICKLE CELL DISEASE: ONGOING RESULTS FROM THE BEACON STUDY EVALUATING THE SAFETY AND EFFICACY OF BEAM-101, THE FIRST BASE-EDITED AUTOLOGOUS CD34+ HSPC ONE-TIME CELL THERAPY (EHA 2025) - P1/2 | "After mobilization with plerixafor, autologous CD34+ HSPCs were collected by leukapheresis and genetically modified with an adenine base editor. Ongoing data from the BEACON study support the safety of BEAM-101 as consistent with busulfan conditioning and autologous hematopoietic stem cell transplant. Pts required 1-2 mobilization cycles and achieved rapid engraftment. Rapid and robust HbF induction and corresponding decrease in HbS was observed in all pts following treatment with BEAM-101, with resolution of anemia."

Clinical • Anemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Neutropenia • Respiratory Diseases • Sickle Cell Disease • CD34

RED BLOOD CELL (RBC) HEALTH AND FUNCTION POST BEAM-101 TREATMENT: MULTIPLE EXPLORATORY BIOMARKERS DEMONSTRATE RHEOLOGY AND SICKLING PARAMETERS COMPARABLE TO SICKLE CELL TRAIT (SCT) (EHA 2025) - P1/2 | "Exploratory RBC biomarker data show that 99% of non-transfused RBCs express HbF as early as M1, with near-complete elimination of RBCs expressing solely HbS post BEAM-101. These data demonstrate reduced RBC sickling and cell adhesion comparable to SCT; improved oxygen affinity, RBC deformability, decreased %DRBCs; and resolution of abnormal RBC morphology post BEAM-101. They support base editing of HBG1/2 promoters as a potentially transformative therapeutic modality for SCD treatment."

Biomarker • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34 • HBG1 • VCAM1

Integrated Editing and Manufacturing Process Design for BEAM-101, an Autologous CD34+ HSPC Therapy for Sickle Cell Disease, Results in Robust Process Yield and Increased HbF Induction (EHA 2025) - P1/2 | "A CRISPR base-edited therapeutic for SCD has been developed through the integration of process development with optimal selection of gene editing methodology. Robust process yield, viability, and editing rates have been demonstrated while the number of mobilization cycles has been reduced. Likewise, the selection of base editing the HBG1/2 promoter yielded superior desired fetal hemoglobin induction compared to a nuclease editing approach."

Genetic Disorders • Hematological Disorders • Sickle Cell Disease • BCL11A • CD34

Beam Therapeutics’ BEAM-101 receives FDA orphan drug status for SCD treatment (Investing.com) - "Beam Therapeutics Inc...has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its investigational cell therapy BEAM-101, aimed at treating sickle cell disease (SCD)....In the ongoing BEACON Phase 1/2 trial, the treatment has shown promising results, including increases in fetal hemoglobin and decreases in sickle hemoglobin, which are crucial for alleviating the symptoms of SCD....Beam Therapeutics expects to dose a total of 30 patients in the BEACON trial by mid-2025."

Orphan drug • Trial status • Sickle Cell Disease

Beam Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights (GlobeNewswire) - "Beam expects to dose 30 patients in the BEACON Phase 1/2 clinical trial of BEAM-101 in adults with severe SCD by mid-2025. The company also plans to present updated data from the BEACON Phase 1/2 trial during EHA 2025 in June."

P1/2 data • Sickle Cell Disease

Beam Therapeutics to Encore Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 in Sickle Cell Disease at 2025 Tandem Meetings of ASTCT and CIBMTR (GlobeNewswire) - P1/2 | N=15 | BEACON (NCT05456880) | Sponsor: Beam Therapeutics Inc. | "Beam Therapeutics Inc...today announced the company will encore data from the BEACON Phase 1/2 clinical trial of BEAM-101 in sickle cell disease in an oral presentation at the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and Center for International Blood and Marrow Transplant Research (CIBMTR) taking place February 12 – 15, 2025, in Honolulu, Hawaii...The safety profile of BEAM-101 was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation. Beam expects to present updated data from the BEACON trial in mid-2025."

P1/2 data • Sickle Cell Disease

Safety and Efficacy of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) for the Treatment of Sickle Cell Disease with Severe Vaso-Occlusive Crises: Results from the Ongoing Phase 1/2 Beacon Study (TCT-ASTCT-CIBMTR 2025) - P1/2 | "After plerixafor mobilization, autologous CD34+ HSPCs were collected by leukapheresis and edited with an adenine base editor. After myeloablative conditioning with busulfan, pts received a single infusion of BEAM-101 (≥3.0 × 10 6 viable CD34+ cells/kg) and are monitored for 24 months (m).."

Clinical • P1/2 data • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Respiratory Diseases • Sickle Cell Disease • CD34

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Beam Therapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • HP

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=15 | Not yet recruiting | Sponsor: Beam Therapeutics Inc.

New P1/2 trial • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • HP

Beam Therapeutics Provides Business and Pipeline Updates and Reports Second Quarter 2021 Financial Results (GlobeNewswire) - "Beam anticipates filing an IND for BEAM-101, which reproduces single base changes seen in individuals with Hereditary Persistence of Fetal Hemoglobin, or HPFH, to potentially protect them from the effects of mutations causing sickle cell disease (SCD), and initiating IND-enabling studies for BEAM-102, which directly corrects the causative mutation in SCD...both in the second half of 2021. Beam is also on track to nominate its first Development Candidate for in vivo base editing of the liver using LNP delivery by the end of 2021....Beam plans to present preclinical data highlighting its approach to using adenine base editing (ABE) for the treatment of sickle cell disease, including updated safety data during an oral session at the Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers Conference."

IND • Preclinical • Hematological Disorders • Sickle Cell Disease