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Sickle Cell disease stories

Nov 10, 2026

Beam Therapeutics base-editing sickle cell therapy succeeds in pivotal trial

Beam Therapeutics announced its base-edited sickle cell therapy met its primary endpoint in a Phase 1/2 clinical trial, potentially positioning it as a third one-time curative therapy for the disease.

Nov 10, 2026·2 sources
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Top claims

  • ▪Beam Therapeutics plans to submit an approval application for risto-cel to the U.S. Food and Drug Administration as early as the end of 2026
  • ▪Beam Therapeutics announced its base-editing treatment ristoglogene autogetemcel met its primary endpoint of freedom from severe vaso-occlusive crises
  • ▪If approved, risto-cel would enter a sickle cell treatment market that includes approved genetic therapies Casgevy and Lyfgenia, alongside hydroxyurea, l-glutamine, blood transfusions, and stem cell transplants

Topics

Clinical trialsFDA regulatory approvalClinical trial endpointsBiotech innovationGene therapyGene editingGenetic diseasesHematology

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