सरल व्याख्या
BEAM-302 does the thing most people imagine when they hear about gene editing, and which almost nothing else on this site actually does: it corrects the mutation. One wrong letter in the alpha-1 antitrypsin gene is changed back to the right one, inside the patient's own liver cells, after a single infusion.
और गहराई से जानें
BEAM-302 is an in vivo base editor delivered by lipid nanoparticle that corrects the SERPINA1 PiZ (E342K) mutation in hepatocytes, simultaneously restoring functional alpha-1 antitrypsin and reducing the misfolded protein that causes liver disease. It produced the first clinical demonstration of correcting a disease-causing point mutation in vivo in humans. A single 60 mg dose raised alpha-1 antitrypsin above the threshold associated with lung protection in all patients dosed, and the first patient in the global pivotal cohort was dosed in 2026.