1 / 1
Fitusiran API is a synthetic small interfering RNA (siRNA) investigated primarily in the field of hemophilia and coagulation disorders. It targets the antithrombin (AT or SERPINC1) gene in the liver to reduce antithrombin production. Researchers use Fitusiran to explore RNA interference (RNAi) mechanisms, liver-specific gene silencing, and novel therapeutic strategies for rebalancing coagulation in hemophilia A and B patients, with or without inhibitors.
Fitusiran functions by silencing the expression of antithrombin, a natural anticoagulant, thereby increasing thrombin generation and promoting clot formation. This mechanism offers a prophylactic treatment approach to reduce bleeding episodes in hemophilia patients. As an API, Fitusiran serves as the active ingredient in long-acting subcutaneous therapies aimed at improving quality of life and reducing treatment burden in bleeding disorders.
Key Takeaway: Fitusiran leverages RNA interference (RNAi) technology to target liver antithrombin production, providing an innovative subcutaneous approach to rebalance coagulation in bleeding disorders.
Fitusiran API is a synthetic small interfering RNA (siRNA) designed to utilize RNA interference (RNAi) mechanisms for target gene silencing.
It specifically targets the antithrombin (AT or SERPINC1) gene in the liver to lower the production of antithrombin.
By silencing antithrombin (a natural anticoagulant), Fitusiran increases thrombin generation, which helps promote blood clot formation to prevent bleeding.
Research focuses on hemophilia A and B patients, both with and without inhibitors, who require rebalanced coagulation therapeutic strategies.
Fitusiran serves as the active pharmaceutical ingredient (API) in long-acting subcutaneous therapies aimed at reducing treatment frequency.
