Beam Therapeutics’ BEAM-101 Demonstrates Differentiated Profile in Sickle Cell Disease

CAMBRIDGE, Mass. – June 13, 2025 – Beam Therapeutics Inc. (Nasdaq: BEAM) today announced new, positive safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with severe sickle cell disease (SCD) with vaso-occlusive crises (VOCs). These updated findings, from 17 treated patients with follow-up up to 15.1 months, were presented at the European Hematology Association (EHA) 2025 Congress in Milan.

BEAM-101 is an investigational precision genetic medicine utilizing base editing to treat SCD. It involves taking a patient’s own CD34+ hematopoietic stem and progenitor cells (HSPCs), editing them to increase fetal hemoglobin (HbF) production, and then reinfusing them. This aims to mimic naturally occurring variants that lead to hereditary persistence of fetal hemoglobin, effectively counteracting the sickling of red blood cells.

Key highlights from the BEACON trial include:

• Robust & Durable HbF Increases: All patients achieved endogenous HbF levels exceeding 60% and a corresponding durable reduction in sickle hemoglobin (HbS) below 40%. Responses were maintained for up to 15 months.
• No VOCs Post-Engraftment: Critically, no patients experienced any investigator-reported VOCs after engraftment.
• Rapid Engraftment & Minimized Transfusions: Patients required a median of just one mobilization cycle, with rapid neutrophil and platelet engraftment (median 16.5 and 19.5 days, respectively), minimizing transfusion needs.
• Improved Red Blood Cell Health: Markers of hemolysis normalized or improved, and exploratory assessments showed red blood cell health and function comparable to sickle cell trait.
• Favorable Safety Profile: The safety profile was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation (HSCT), with no new safety signals.

“These promising and consistent new data from the BEACON trial continue to reinforce the potential of this gene therapy to fulfill this unmet need”, said Dr. Ashish Gupta, an investigator in the BEACON trial.

John Evans, CEO of Beam, added that the data further builds on BEAM-101’s potential “to deliver a transformative treatment for patients with SCD,” noting differentiated outcomes and consistently strong manufacturing success.