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Beam Therapeutics Says Risto-Cel Meets Primary Endpoints in Sickle Cell Disease Trial

Beam Therapeutics Says Risto-Cel Meets Primary Endpoints in Sickle Cell Disease Trial

MT newswireMT newswire2026/11/10 13:59
09:59 AM EST, 11/10/2026 (MT Newswires) -- Beam Therapeutics (BEAM) said Tuesday its Beacon phase 1/2 study of ristoglogene autogetemcel, or risto-cel, in patients with sickle cell disease met its primary efficacy and safety endpoints at a prespecified interim analysis. The study showed patients were free from severe vaso-occlusive crises for at least 12 consecutive months, while all secondary efficacy endpoints were also met, including freedom from inpatient hospitalization for severe vaso-occlusive crises for at least a year, the company said. As of the Oct. 20 data cutoff, 49 patients had been treated and 31 had completed the required follow-up for the primary endpoint. Beam said the safety findings were consistent with previously reported data. The company said it expects to submit a biologics license application for risto-cel to the US Food and Drug Administration as early as the end of 2026. Shares of Beam were down 2.8% in Tuesday trading. Price: 26.00, Change: -1.02, Percent Change: -3.77
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华尔街见闻•2026/11/10 18:41
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