Hemophilia B is a promising target for gene therapy because it is caused by a single gene mutation

Gene therapies for hemophilia B currently being studied use AAV vector technology to deliver a functional gene, with the goal that a patient’s liver can generate its own factor IX (FIX). The goal of therapy is that a single, one-time administration could provide years of bleed protection.

About CSL Behring

CSL Behring is a global leader in developing and delivering high-quality medicines that treat people with rare and serious diseases. Our treatments offer promise for people in more than 100 countries living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas.