Spark Therapeutics offers a glimpse of efficacy in first two hemophilia A gene therapy patients
With its lead gene therapy under FDA review, Spark Therapeutics $ONCE is now unveiling the first encouraging responses on the hemophilia A front.
With analysts looking for a 12%-plus Factor VIII response, the company says that investigators tracked an 11% and 14% stabilized rise in Factor VIII activity in the first two patients who were given the therapy in the dose-escalation study.
Key to this part of the process, researchers say they have seen no Factor VIII inhibitors appear, no thrombotic events, no spontaneous bleeds and no need to use corticosteroids on the patients, who have been tracked for 23 and 12 weeks.
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