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Start of Phase I/II Gene Therapy Clinical Trial for Hemophilia B
Date:3/10/2010

the absence of functional clotting Factor IX as a result of mutations in the gene encoding this protein. The factor IX gene is located on the X chromosome. It is an X-linked recessive trait, which explains why only males are usually affected.

Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almost always in males. The total number of patients in Europe and the USA together is estimated to be between 35,000 and 40,000.

Currently, frequent intravenous administrations of recombinant Factor IX are required to stop or prevent bleeding. Protein replacement therapy is costly, cumbersome, and does not completely prevent bleeding.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using AAV as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. This safe and efficacious proprietary platform offers
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SOURCE Amsterdam Molecular Therapeutics B.V
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