ALN-F5

ALN-F5 is an investigational RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals for the treatment and prevention of thrombosis. It consists of a small interfering RNA (siRNA) designed to target the messenger RNA (mRNA) of Coagulation Factor V (F5) in the liver. The drug utilizes Alnylam's Enhanced Stabilization Chemistry (ESC) and is conjugated to a N-acetylgalactosamine (GalNAc) moiety, which enables targeted delivery to hepatocytes via the asialoglycoprotein receptor. By mediating the catalytic degradation of F5 mRNA, ALN-F5 significantly reduces the synthesis and circulating levels of the Factor V protein, a critical cofactor in the prothrombinase complex of the coagulation cascade. This reduction in Factor V levels is intended to dampen thrombin generation and lower the risk of venous thromboembolism (VTE), particularly in high-risk populations such as those with the Factor V Leiden mutation. ALN-F5 is designed for subcutaneous administration, offering a potentially long-acting alternative to traditional anticoagulants.

Other names

factor 5a small interfering RNAfactor5a small interfering RNAfactor-5a small interfering RNAFactor V siRNAF5-siRNAF-5-siRNAF 5-siRNA