Beam Therapeutics Reports Durable Efficacy and Safety for Risto-cel in Sickle Cell Disease Trial

Reuters12-06 21:00

Beam <a href="https://laohu8.com/S/LENZ">Therapeutics</a> Reports Durable Efficacy and Safety for Risto-cel in Sickle Cell Disease Trial

Beam Therapeutics Inc. announced updated safety and efficacy data from its BEACON Phase 1/2 clinical trial evaluating ristoglogene autogetemcel (risto-cel), formerly known as BEAM-101, in patients with sickle cell disease $(SCD)$ experiencing severe vaso-occlusive crises. The results, presented at the 67th American Society of Hematology $(ASH)$ Annual Meeting and Exposition, showed that among 31 adult and adolescent SCD patients treated with risto-cel, there was a mean hemoglobin F (HbF) induction of over 60%, hemoglobin S (HbS) reduction to below 40%, and resolution of anemia durable for up to 20 months. The safety profile was consistent with busulfan conditioning, autologous hematopoietic stem cell transplantation, and underlying SCD. Additionally, a tiered fixed-dose plerixafor mobilization regimen was found to yield higher CD34+ cell counts and more efficient stem cell collection compared to traditional weight-based dosing.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Beam Therapeutics Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9597866-en) on December 06, 2025, and is solely responsible for the information contained therein.

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The results, presented at the 67th American Society of Hematology  Annual Meeting and Exposition, showed that among 31 adult and adolescent SCD patients treated with risto-cel, there was a mean hemoglobin F  induction of over 60%, hemoglobin S  reduction to below 40%, and resolution of anemia durable for up to 20 months. The safety profile was consistent with busulfan conditioning, autologous hematopoietic stem cell transplantation, and underlying SCD. 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The results, presented at the 67th American Society of Hematology $(ASH)$ Annual Meeting and Exposition, showed that among 31 adult and adolescent SCD patients treated with risto-cel, there was a mean hemoglobin F (HbF) induction of over 60%, hemoglobin S (HbS) reduction to below 40%, and resolution of anemia durable for up to 20 months. The safety profile was consistent with busulfan conditioning, autologous hematopoietic stem cell transplantation, and underlying SCD. Additionally, a tiered fixed-dose plerixafor mobilization regimen was found to yield higher CD34+ cell counts and more efficient stem cell collection compared to traditional weight-based dosing.\n      \n\nDisclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. 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