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

once, will introduce the functional gene for the Factor IX protein into the patient's liver cells with the goal to restore blood clotting functionality long-term. In pre-clinical studies, Factor IX gene therapy resulted in long-term production of Factor IX protein at a therapeutically significant level after a single administration. If this approach is successful, the long term efficacy of one time administered hemophilia B gene therapy is expected to be perceived as a significant advance over the current regular dosing of recombinant Factor IX. In addition, the efficacy profile of this gene therapy is anticipated to exceed that of current therapy, as the gene therapy should lead to stable Factor IX levels whereas current recombinant protein treatment causes peaks and troughs. It is hoped that hemophilia B gene therapy, therefore, can potentially replace all recombinant Factor IX products.

The UK Medicines and Healthcare products Regulatory Agency as well as the US Food and Drug Administration have approved the current trial.

About the Disease

Hemophilia B is characterized by severe episodes of external and internal bleeding, resulting in significant morbidity. The episodes cause long-term damage, for instance to the joints, and may be fatal if they occur in the brain. The defect in blood clotting in hemophilia B is caused by
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SOURCE Amsterdam Molecular Therapeutics B.V
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