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Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present updated biomarker data from the BEACON Phase 12 clinical trial of ristoglogene autogetemcel (risto-cel) in sickle cell disease (SCD) at the European Hematology Association 2026 Congress (EHA2026), taking place June 11-14, 2026, in Stockholm, Sweden. Risto-cel is an investigational autologous cell therapy with a potential best-in-class profile for the treatment of SCD.
"The comprehensive analyses being presented at EHA2026 further underscore the potential of risto-cel to correct key drivers of sickle cell disease pathology," said Amy Simon, M.D., chief medical officer of Beam. "The data continue to support the ability of risto-cel to restore red blood cell health and function, including improvements in sickling and rheologic parameters comparable to sickle cell trait, while also reinforcing the relationship between fetal hemoglobin induction and improved clinical outcomes in sickle cell disease. Together, these findings contribute to the growing body of evidence supporting risto-cel's potential differentiated profile for patients living with severe sickle cell disease."
Details are as follows:
Title: Ristoglogene Autogetemcel Treatment Restored Red Blood Cell Health and Function in Patients with Sickle Cell Disease, with Sickling and Rheology Parameters Comparable to Sickle Cell Trait
Abstract: PS2332
Poster Session: Poster Session 2
Session Time: Saturday, June 13, 2026, 6:45 - 7:45 p.m. CEST
Presenter: Priya S. Chockalingam, Ph.D., Beam Therapeutics
Title: Correlation Between Fetal Hemoglobin and Clinical Outcomes in Sickle Cell Disease: A Model-Based Meta-Analysis
Abstract: PB4134
Format: Publication-only in the official Abstract Book, EHA Library and EHA2026 Congress platform
Author: Bahru Habtemariam, Pharm.D., Beam Therapeutics
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, 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 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 potential 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.
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 programs and present data from ongoing clinical trials; the clinical trial designs and expectations for risto-cel; our expected presentations at upcoming medical conferences; our anticipated regulatory interactions and filings; and our ability to develop lifelong, 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 initiate or continue 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, including 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, 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
