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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