Beam Therapeutics to Highlight New Data from BEAM-101 Program in Sickle Cell Disease at European Hematology Association (EHA) 2025 Congress
Beam Therapeutics (NASDAQ: BEAM) will present new data from its BEACON Phase 1/2 clinical trial of BEAM-101, a base-edited cell therapy for sickle cell disease (SCD), at the European Hematology Association 2025 Congress in Milan. The presentation will include updated safety and efficacy data from 17 patients treated with BEAM-101, a one-time treatment for SCD patients with severe vaso-occlusive crises (VOCs).
The company will showcase four presentations at EHA2025, focusing on BEAM-101's clinical results, red blood cell health post-treatment, manufacturing process, and biomarker findings. Beam will host an investor webcast on June 13, 2025, at 4:00 p.m. ET to review the key presentations.
Beam Therapeutics (NASDAQ: BEAM) presenterà nuovi dati del suo studio clinico di fase 1/2 BEACON su BEAM-101, una terapia cellulare con editing di basi per la malattia falciforme (SCD), al Congresso 2025 dell'Associazione Europea di Ematologia a Milano. La presentazione includerà dati aggiornati su sicurezza ed efficacia di 17 pazienti trattati con BEAM-101, un trattamento unico per pazienti con SCD affetti da crisi vaso-occlusive gravi (VOC).
L'azienda presenterà quattro interventi a EHA2025, concentrandosi sui risultati clinici di BEAM-101, la salute dei globuli rossi dopo il trattamento, il processo di produzione e le scoperte sui biomarcatori. Beam terrà un webcast per investitori il 13 giugno 2025 alle 16:00 ET per rivedere le presentazioni principali.
Beam Therapeutics (NASDAQ: BEAM) presentará nuevos datos de su ensayo clínico de fase 1/2 BEACON sobre BEAM-101, una terapia celular con edición de bases para la enfermedad de células falciformes (SCD), en el Congreso 2025 de la Asociación Europea de Hematología en Milán. La presentación incluirá datos actualizados de seguridad y eficacia de 17 pacientes tratados con BEAM-101, un tratamiento único para pacientes con SCD que sufren crisis vasooclusivas severas (VOC).
La compañía realizará cuatro presentaciones en EHA2025, enfocándose en los resultados clínicos de BEAM-101, la salud de los glóbulos rojos tras el tratamiento, el proceso de fabricación y los hallazgos de biomarcadores. Beam organizará una transmisión web para inversores el 13 de junio de 2025 a las 4:00 p.m. ET para repasar las presentaciones clave.
Beam Therapeutics (NASDAQ: BEAM)는 BEAM-101에 대한 BEACON 1/2상 임상시험의 새로운 데이터를 2025년 유럽 혈액학회(EHA) 총회에서 발표할 예정입니다. BEAM-101은 겸상 적혈구 질환(SCD)을 위한 베이스 에디팅 세포 치료제입니다. 발표에는 중증 혈관폐쇄 위기(VOC)를 겪는 SCD 환자 17명에게 단회 투여된 BEAM-101의 안전성과 효능에 대한 최신 데이터가 포함됩니다.
회사는 EHA2025에서 BEAM-101의 임상 결과, 치료 후 적혈구 건강, 제조 공정 및 바이오마커 발견에 관한 4건의 발표를 진행할 예정이며, 2025년 6월 13일 오후 4시(동부시간)에 주요 발표 내용을 검토하는 투자자 웹캐스트를 개최합니다.
Beam Therapeutics (NASDAQ : BEAM) présentera de nouvelles données issues de son essai clinique de phase 1/2 BEACON sur BEAM-101, une thérapie cellulaire par édition de bases pour la drépanocytose (SCD), lors du Congrès 2025 de l'Association Européenne d'Hématologie à Milan. La présentation inclura des données actualisées sur la sécurité et l'efficacité provenant de 17 patients traités avec BEAM-101, un traitement unique pour les patients atteints de SCD souffrant de crises vaso-occlusives sévères (VOC).
L'entreprise proposera quatre présentations à l'EHA2025, mettant l'accent sur les résultats cliniques de BEAM-101, la santé des globules rouges après traitement, le processus de fabrication et les découvertes sur les biomarqueurs. Beam organisera un webcast pour les investisseurs le 13 juin 2025 à 16h00 ET afin de passer en revue les présentations clés.
Beam Therapeutics (NASDAQ: BEAM) wird neue Daten aus der BEACON Phase-1/2-Studie zu BEAM-101, einer baseneditierten Zelltherapie für die Sichelzellanämie (SCD), auf dem European Hematology Association 2025 Kongress in Mailand vorstellen. Die Präsentation umfasst aktualisierte Sicherheits- und Wirksamkeitsdaten von 17 Patienten, die mit BEAM-101 behandelt wurden, einer einmaligen Behandlung für SCD-Patienten mit schweren vaso-okklusiven Krisen (VOC).
Das Unternehmen wird auf der EHA2025 vier Präsentationen halten, die sich auf die klinischen Ergebnisse von BEAM-101, die Gesundheit der roten Blutkörperchen nach der Behandlung, den Herstellungsprozess und Biomarker-Ergebnisse konzentrieren. Beam veranstaltet am 13. Juni 2025 um 16:00 Uhr ET ein Investoren-Webcast, um die wichtigsten Präsentationen zu besprechen.
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Insights
Beam Therapeutics' upcoming presentation of BEAM-101 clinical data for sickle cell disease represents significant progress in their base editing treatment program.
Beam Therapeutics is preparing to share new clinical data for their base editing cell therapy BEAM-101 at the upcoming European Hematology Association Congress. The presentation will include updated safety and efficacy results from 17 patients with sickle cell disease (SCD) enrolled in their BEACON Phase 1/2 trial - a meaningful sample size for this stage of development.
What makes this particularly significant is that BEAM-101 represents one of the first base editing therapies to reach clinical trials. Unlike CRISPR, which cuts DNA, base editing precisely changes individual DNA bases without double-strand breaks, potentially offering improved safety profiles. For SCD patients, this approach targets the root genetic cause of the disease rather than merely treating symptoms.
The company is also presenting additional data on red blood cell health post-treatment, with biomarkers showing rheology and sickling parameters comparable to sickle cell trait - this suggests the therapy may effectively modify disease pathophysiology to a milder form. Their manufacturing presentation indicates they've achieved robust process yields with increased fetal hemoglobin induction, critical for commercial viability.
SCD affects millions globally, causing painful vaso-occlusive crises, organ damage, and reduced lifespan. Current standard treatments are limited, with the only curative option being stem cell transplantation, which has significant limitations. A one-time cell therapy like BEAM-101 could represent a transformative advance if the data confirms durable efficacy with manageable safety profiles. This upcoming data presentation will be a critical milestone in evaluating whether base editing can deliver on its therapeutic promise for this devastating genetic condition.
Presentation to Include Updated Data from 17 Sickle Cell Disease Patients in the Ongoing BEACON Phase 1/2 Clinical Trial Evaluating Safety and Efficacy of BEAM-101
Beam to Host Investor Webcast on Friday, June 13, 2025, at 4:00 p.m. ET
CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present new data from the BEACON Phase 1/2 clinical trial of BEAM-101 in sickle cell disease (SCD) at the European Hematology Association 2025 Congress (EHA2025), taking place June 12-15, 2025, in Milan, Italy. BEAM-101 is an investigational genetically modified ex vivo cell therapy for the treatment SCD with severe vaso-occlusive crises (VOCs).
“We are excited to share an updated safety and efficacy dataset from 17 patients in the BEACON Phase 1/2 clinical trial at EHA2025, as we continue to build on our understanding of this potentially transformative, one-time treatment for people living with SCD,” said Amy Simon, M.D., chief medical officer of Beam. “SCD affects millions of people worldwide, and we are committed to delivering a potential lifelong treatment that addresses the root cause of this debilitating condition. These new data, along with additional insights from our other presentations at EHA2025, further enhance our understanding of the disease, support a differentiated profile of BEAM-101 and underscore the promise of base editing in creating precision therapies for patients.”
Presentation details are as follows:
BEAM-101 Presentations:
Title: 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
Abstract: PF1151
Poster Session: Poster Session 1
Session Time: Friday, June 13, 2025, 6:30 - 7:30 p.m. CEST
Presenter: Ashish Gupta, M.D., MPH, University of Minnesota
Title: 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)
Abstract: PF1155
Poster Session: Poster Session 1
Session Time: Friday, June 13, 2025, 6:30 - 7:30 p.m. CEST
Presenter: Priya S. Chockalingam, Ph.D., Beam Therapeutics
Title: 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
Abstract: PF1165
Poster Session: Poster Session 1
Session Time: Friday, June 13, 2025, 6:30 - 7:30 p.m. CEST
Presenter: Paul Kopesky, Ph.D., Beam Therapeutics
Collaborator Presentation:
Title: Identifying Potential Biomarkers of Clinical Relevance in Sickle Cell Trait: Interim Findings from the National Alliance of Sickle Cell Centers AUNT Study
Abstract: PF1174
Poster Session: Poster Session 1
Session Time: Friday, June 13, 2025, 6:30 - 7:30 p.m. CEST
Presenter: Julie Kanter, M.D., the University of Alabama at Birmingham
EHA Investor Webcast Information
Beam will host a conference call and webcast on Friday, June 13, 2025, at 4:00 p.m. ET to review key presentations from this year’s EHA meeting. A live webcast of the presentation will be available under "Events" in the Investors section of the company's website at www.beamtx.com, and a replay will be available shortly after the event.
About BEAM-101
BEAM-101 is an investigational genetically modified cell therapy for the treatment of severe 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 promotor regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking 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 BEAM-101 is being evaluated in the ongoing BEACON Phase 1/2 study, an open-label, single-arm, multicenter trial in adult patients with SCD with severe vaso-occlusive crises (VOCs).
About Sickle Cell Disease (SCD)
Sickle cell disease, 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. Sickle cell disease is the most common inherited blood disorder in the United States, affecting an estimated 100,000 individuals within the United States 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 life-long cures to patients suffering from serious diseases.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including with respect to SCD; our plans, and anticipated timing, to advance our BEAM-101 program, including the clinical trial designs and expectations for BEAM-101; our plans to present data at EHA2025; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to advance human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates or the delivery modalities we rely on to administer them may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Report on Form 10-K for the quarter ended March 31, 2025, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.
Contacts:
Investors:
Holly Manning
Beam Therapeutics
hmanning@beamtx.com
Media:
Josie Butler
1AB
josie@1abmedia.com
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