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
The gene therapies for hemophilia B that are in clinical trials use AAV vector technology to deliver a functional—or working—gene, so the liver can generate its own protective levels of factor IX (FIX). A single, one-time administration could provide years of bleed protection.
Learn more about AAV vectors
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With a history spanning over 100 years, CSL Behring has been focused on serving patients’ needs by using the latest technologies to develop and deliver innovative therapies. Our advances in hemophilia treatment, and serving that community, have spanned decades. It’s all part of delivering on our promise to transform the lives of people around the world living with serious and rare diseases.
