First clinical data for BioMarin hemo A gene therapy
At its R&D day on Wednesday, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported preliminary data from the first eight patients with severe hemophilia A who received a single dose of IV BMN 270 in an ongoing dose-escalation Phase I/II trial of the gene therapy. According to BioCentury's BCIQ database, BMN 270 is the only clinical-stage gene therapy program for hemophilia A.
BioMarin said disease severity has improved to either mild hemophilia A or normal in five of the six patients who received the highest dose of BMN 270, while one has improved to moderate disease, according to World Federation of Hemophilia (WFH) criteria. Those patients' Factor VIII activity levels were measured 5-16 weeks after treatment. One patient receiving an intermediate dose had moderate disease after 16 weeks, and one receiving a low dose had severe disease after 20 weeks. ...