Spark carefully records a safe step forward for closely-watched hemophilia gene therapy
Four months after rattling investors with evidence of an immune reaction to its gene therapy, Spark Therapeutics $ONCE is updating its data on the Phase I/II study of its hemophilia B treatment, offering fresh evidence that researchers were able to resolve the issue without losing the treatment effect.
Spark also noted that with all 10 patients past at least 12 weeks of therapy — and two past the one-year mark — the once-and-done approach to hemophilia had almost — though not quite — wiped out infusions for the group. And the annual bleed rate was cut 96%.
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