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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
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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