Pioneering gene therapy patients stay on track, boosting Spark’s hemophilia B program
After rattling investors with early signs of an immune reaction in a couple of cases as well as an unexpected infusion for a suspected knee bleed, Spark Therapeutics $ONCE says that its early Phase I/II study for its hemophilia B gene therapy is staying on track, slashing the rate of annual infusions and the bleed rate among the 10 patients on the pioneering therapy SPK-9001.
The annual infusion rate has new dropped 99%, down to a mean of 1 compared to 67.5 ahead of treatment. Five of the 10 are now past the one-year mark since their treatment, with no bleeding issues. The group of 10 posted an average bleed rate of 0.4 compared to 11.1 ahead of once-and-done therapy.
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