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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