Goals of gene therapy for hemophilia B

Gene therapy for hemophilia B aims to deliver lasting bleed protection for years

The goals of gene therapy being studied for hemophilia B are:


To provide the long-term benefit of sustained factor
activity levels from a single administration of treatment

To reduce or even eliminate bleeding and the need
for regular, long-term prophylaxis

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Hemophilia B is an appropriate target for treatment with gene therapy because it is caused by a single gene mutation. Gene therapies under investigation aim to provide a working copy of the F9 gene.

AAV gene therapies differ in the way the vector is selected, and can affect their safety and effectiveness. Some AAVs may be used even with people who have a small amount of pre-existing antibodies to that vector.

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In clinical trials, AAV gene therapy in hemophilia B showed stable FIX activity for over 8 years of follow-up, with sustained decreases in annualized bleed rate (ABR) and FIX replacement therapy.

Gene therapy clinical trials for hemophilia B are still ongoing; currently, none are FDA ‑ approved.

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