Beam Therapeutics is developing a gene-editing therapy called BEAM-101 for sickle cell disease using base-editing technology. The therapy aims to increase the production of functional hemoglobin and improve the shape of red blood cells. Early results from the Phase 1/2 study show promising outcomes in patients, with increased levels of fetal hemoglobin and fewer sickle-shaped cells. However, one patient died due to complications related to the chemotherapy drug busulfan used in preparing patients for treatment. Beam is also working on a less toxic preconditioning option called ESCAPE, which could potentially eliminate the need for busulfan.
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