Pharma/Bio-tech Update: BioMarin Announces Selection of Factor VIII Gene Therapy Drug
January 13, 2014; Posted by: WeBleed staff
BioMarin Pharmaceuticals to begin research and studies on new hemophilia A drug.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. Now they can add hemophilia to their list.
In a recent release, BioMarin announced their selection of a factor 8 (hemophilia A) vector for their upcoming studies and trials for a new hemophilia drug. The main reason for this new development has been due to an ongoing gene therapy program for hemophilia A that was originally licensed from the University College London (UCL) and St. Jude Children’s research Hospital in February 2013 and has since been developed at BioMarin’s facilities.
“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, in a recent press release. “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.”
WeBleed will update the progress on this new development as information becomes available.
Photo Credit – BioMarin Pharmaceuticals
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