The therapeutic landscape for PNH has significantly evolved, with an increasing number of complement inhibitors now available, including the C5 inhibitors eculizumab, ravulizumab, and crovalimab, the C3/C3b inhibitor pegcetacoplan, and the AP complement inhibitors iptacopan and danicopan. The gene discussed is C5; the disease is paroxysmal nocturnal hemoglobinuria.