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ひとことで言うと
Beam was built around base editing, the technique its co-founder invented for changing one DNA letter into another without cutting the double helix. Its sickle cell therapy has finished dosing in trials with a licence application signalled for as early as the end of 2026, and its alpha-1 antitrypsin programme produced the first correction of a disease-causing mutation inside a living person.
Beam Therapeutics was founded in 2017 by David Liu, Feng Zhang and J. Keith Joung. Its portfolio spans ex vivo base editing (risto-cel for sickle cell disease) and in vivo base editing (BEAM-302 for alpha-1 antitrypsin deficiency, BEAM-301 for glycogen storage disease type 1a). BEAM-103, an anti-CD117 antibody, aims to replace genotoxic busulfan conditioning — potentially removing the largest source of toxicity in ex vivo cell therapy.
