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BioMarin Announces Selection Of Factor VIII Gene Therapy Drug Development Candidate BMN 270 For The Treatment Of Hemophilia A

SAN RAFAEL, Calif., Jan. 13, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has selected an AAV-factor VIII vector, BMN 270, to develop for the treatment of hemophilia A and has initiated IND-enabling studies. BioMarin expects to initiate clinical studies with BMN 270 in early 2015. The Company's gene therapy program for hemophilia A was originally licensed from University College London (UCL) and St. Jude Children's research Hospital in February 2013 and has since been developed at BioMarin's facilities.   

"In our hemophiliac factor VIII deficient mouse models, BMN 270 restored factor VIII plasma concentrations to levels projected to be adequate for normal clotting in humans," said Barrie Carter, Ph.D.,  Vice President, Vector Biology, of BioMarin. "In the past eleven months since we acquired the AAV-factor VIII technology from UCL and  St. Jude, we have evaluated a large number of candidate vectors for treating hemophilia A, and we are pleased to have selected BMN 270 as BioMarin's next IND candidate."

"Gene therapy is emerging as a powerful way to treat genetic disorders and is complementary to our current suite of commercial products and research programs," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Hemophilia is an attractive target for gene therapy as factor levels in the blood serve as good biomarkers, relatively low factor levels are required for a clinically important benefit and the current standard of care of multiple intravenous infusions per week is quite onerous for patients. We remain committed to maintaining a rich pipeline with the goal of filing an IND every twelve to eighteen months."

Professor Amit Nathwani, Ph.D., at University College London added, "UCL is a world leader in the biomedical sciences, with an unremitting commitment to outstanding research and translation into healthcare benefits for patients. We are pleased to collaborate with BioMarin in this exciting field of gene therapy for hemophilia and keen to see a revolution in treatment for this debilitating condition."

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