fibrinogen alpha-chain-targeting small interfering RNA lipid nanoparticle

This research-stage RNA therapeutic consists of small interfering RNA (siRNA) encapsulated within lipid nanoparticles (LNPs) designed to target the fibrinogen alpha chain (FGA). The drug works by inducing the hepatic knockdown of Fga mRNA via the RNA interference (RNAi) pathway, which leads to a controllable and dose-dependent depletion of circulating plasma fibrinogen levels. Preclinical studies have demonstrated that this approach can significantly reduce clot strength while maintaining basic hemostasis, suggesting potential utility in treating thrombosis without the high bleeding risk associated with traditional anticoagulants. Additionally, the compound has shown efficacy in suppressing inflammatory responses in models of endotoxemia and reducing the growth and metastatic potential of tumors, particularly in pancreatic ductal adenocarcinoma (PDAC), where fibrin deposits in the tumor microenvironment contribute to disease progression.

Other names

fibrinogen alpha chain siRNA lipid nanoparticleFGA-targeting siRNA LNPsiFga-lipid nanoparticle