Phase 1 Results with Fitusiran Published in “The New England Journal of Medicine”

Phase 1 Results with Fitusiran Published in “The New England Journal of Medicine”

We have published results from our Phase 1 study with fitusiran in The New England Journal of Medicine, in a paper titled, “Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.”

The published results from Parts A-C of the Phase 1, multicenter, international, open-label single- and multiple-ascending dose escalation study in healthy volunteers and patients with hemophilia A and B without inhibitors. This is the first publication of safety, tolerability and initial clinical activity data for fitusiran in patients with hemophilia A and B.



SIGN UP FOR PATIENT CONNECT

Receive updates on our investigational therapies and clinical trials.