This site is intended only for US Healthcare Professionals

Timeline Helix Image that scrolls down the page
Timeline image with milestones in hemophila treatment advances Timeline image with milestones in hemophila treatment advances
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.

Keep up with the Evolving science OF GENE THERAPY FOR HEMOPHILIA B

Sign up for news of advances in gene therapy for hemophilia B.

SIGN UP NOW >
WHAT TOPICS ON GENE THERAPY FOR HEMOPHILIA B INTEREST YOU



Please select an answer
THANKS FOR LETTING US KNOW. HAVE YOU SIGNED UP TO RECEIVE THE LATEST UPDATES IN GENE THERAPY FOR HEMOPHILIA B YET?
REGISTER NOW >

How can gene therapy fit into your practice?