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Why BioMarin’s gene therapy initially targets one type of hemophilia instead of another
San Francisco Business Times

When researchers at University College London used a gene therapy to effectively cure six patients of hemophilia B, BioMarin Pharmaceutical Inc. took notice.

But the San Rafael company (NASDAQ: BMRN), which licensed in the therapy in February 2013 from the college and St. Jude Children’s Research Hospital, hasn’t stuck to the script. Instead of targeting hemophilia B, BioMarin scientists focused on hemophilia A — and the reasons cut across business and science.