UniQure may be getting all the ASH attention for hemophilia B, but Pfizer is trying to carve out a niche in hemophilia A.
The pharma giant and its gene editing partner Sangamo announced long-term data from their Phase I/II gene therapy, showing that all five patients that have been on the therapy have seen “sustained factor VIII levels.” The median factor VIII levels were 56.9%, with the average coming to 70.4%.
No patient required infusions or suffered a bleedin
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