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Every step has been evolving the
science of gene therapy in hemophilia B

Timeline image with milestones in hemophila treatment advances

1970First patients ever receive gene therapy

1997First rFIX products approved by FDA

1999First gene therapy trial in Hemophilia B

2018Late-stage trials for gene therapy in hem B underway

Every step has been
evolving the science
of gene therapy in
hemophilia B

We’re working to make gene therapy
a reality for you and your patients with hemophilia B

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.

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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.