Fig. 1
From: Advances in the management of haemophilia: emerging treatments and their mechanisms
Diagram illustrating the mechanism of action of Fitusiran/ALN-AT3. GalNAc-siRNA conjugate enters hepatocytes and binds with a ribonucleoprotein to form the RNA-induced silencing complex (RISC) [56]. Once formed, it targets and degrades the SERPINC1 mRNA which encodes antithrombin, effectively silencing the gene. The consequent inhibition of antithrombin production inhibits its down-regulatory effects on FIXa, FXa and thrombin (FIIa). Subsequent increased FIIa generation will alleviate the bleeding phenotype in haemophilia A and haemophilia B. siRNA small interfering ribonucleic acid; RISC RNA-induced silencing complex; mRNA messenger ribonucleic acid; FIX factor IX; FX factor X; FII factor II/thrombin; FIXa activated factor FIX; FXa activated factor X; FIIa activated factor