ALN-CFB

ALN-CFB (also known as REGN4461) is an investigational small interfering RNA (siRNA) therapeutic designed to inhibit the production of Complement Factor B (CFB), a key protease in the alternative complement pathway. Developed through a strategic collaboration between Alnylam Pharmaceuticals and Regeneron Pharmaceuticals, the drug utilizes Alnylam's Enhanced Stabilization Chemistry (ESC) GalNAc-conjugate technology to facilitate targeted delivery to the liver following subcutaneous administration. By silencing CFB mRNA, ALN-CFB reduces the levels of CFB protein circulating in the blood, thereby limiting the activation of the alternative pathway. This mechanism is primarily being evaluated for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients who exhibit persistent anemia and extravascular hemolysis despite treatment with standard-of-care C5 inhibitors. The drug is also being explored for other complement-mediated disorders where alternative pathway dysregulation plays a central role.

Other names

siRNA targeting Complement Factor BComplement Factor B siRNA