Vertex Pharmaceuticals and CRISPR Therapeutics

New Capabilities

Markets Casgevy, the first approved CRISPR therapy for sickle cell

Beam Therapeutics said Tuesday that its sickle cell treatment, risto-cel, met the main goal of a late-stage trial: patients stayed free of severe pain crises for at least 12 months. It is the first positive late-stage result for base editing, a gene-editing method that changes individual DNA letters without cutting the strand. Beam plans to file for FDA approval as early as the end of 2026.

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