Fitusiran

Fitusiran (ALN-AT3SC) is a small interfering RNA compound designed to selectively bind to antithrombin (AT) messenger RNA in the liver, leading to decreased AT production. By doing so, Fitusiran enhances thrombin generation, making it a promising candidate for hemophilia research.

GalNAc unconjugated/naked Fitusiran 是一种没有偶联 GalNAc 的小干扰 RNA，专门针对抗凝血酶(AT)信使 RNA，以降低肝脏中 AT 的产生。GalNAc unconjugated/naked Fitusiran 增加凝血酶的生成，并可用于血友病的研究。