Fitusiran

Fitusiran (API)

Research Application:
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.

Function:
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.