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Why AAV gene therapy fits hemophilia B

Target the root of the problem with a single administration

AAV-based gene therapies
are well-suited for hemophilia B

Hemophilia B is an ideal target for AAV-based gene therapy because it is caused by genetic mutations in a single gene small enough to fit inside a standard AAV vector. In clinical trials, AAV-based gene therapies in hemophilia B are currently being investigated to determine if patients can display durable endogenous factor IX (FIX) activity.

Naturally occurring AAVs have serotype-specific tropism for particular cells and tissues, including the liver where FIX is produced, making AAV vectors a focus of study in gene therapy for hemophilia B.

Goals of gene therapy
for hemophilia B

The goals of AAV gene therapy for hemophilia B are to provide long-term benefits, with sustained factor activity levels from a single administration of treatment, to reduce spontaneous bleeding and the need for chronic prophylaxis.

Take a deeper Dive

Watch Dr. George discuss why hemophilia B is a good candidate for gene therapy.

Dr George discusses why hemophilia B is a good candidate for gene therap

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