Biopharmaceuticals company BioMarin Pharmaceutical Inc (nasdaqgs:BMRN) said on Thursday that it has entered into an agreement to license a Factor VIII gene therapy programme for the potential therapeutic treatment of haemophilia A from University College London (UCL) and St Jude Children's Research Hospital.
Following the selection of a development candidate this year, BioMarin Pharmaceutical plans to initiate and complete IND-enabling toxicology studies next year and initiate proof of concept human studies by the end of 2014.
BioMarin Pharmaceutical's license and commitment to support the research programme was made possible by UCL Business, UCL's wholly-owned technology transfer company, working with Professor Amit Nathwani of the UCL Cancer Institute.
"Gene therapy is emerging as a powerful and viable way to treat genetic disorders and is complementary to its current suite of commercial products and research programmes," said Jean-Jacques Bienaime, CEO of BioMarin. "Haemophilia 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 in severe patients and the current standard of care of intravenous infusions three times a week is quite onerous."
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