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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