QR059

QR059 is an orally bioavailable small molecule inhibitor of Complement factor B (CFB), currently under development by Createrna Development for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a rare hematologic disorder characterized by complement-mediated intravascular and extravascular hemolysis. QR059 selectively targets the alternative pathway of the complement system by binding to Factor B, which is essential for the formation of the C3 convertase (C3bBb). By inhibiting this amplification loop, QR059 prevents the downstream activation of the complement cascade that leads to red blood cell destruction. As an oral therapy, it is designed to offer a more convenient administration route compared to traditional injectable complement inhibitors. The drug is currently in Phase 2 clinical development.