#ASH17: Spark trig­gers a back­lash as ear­ly he­mo­phil­ia A gene ther­a­py da­ta looks shaky

Fol­low­ing up on its im­pres­sive work on a new gene ther­a­py for he­mo­phil­ia B, Spark Ther­a­peu­tics $ONCE has of­fered a peek at its cards on the ear­ly da­ta from a sep­a­rate pro­gram it has on he­mo­phil­ia A. And it’s clear that there’s a lot of work to be done.

Look­ing over a tiny group of pa­tients with vary­ing de­grees of ex­po­sure to the fac­tor VI­II gene car­ry­ing SPK-8011, in­ves­ti­ga­tors tracked a range of re­spons­es rang­ing from sin­gle to dou­ble-dig­it re­spons­es in fac­tor VI­II lev­els. And com­ing af­ter some sol­id da­ta from Bio­Marin, which has the lead­ing gene ther­a­py pro­gram in he­mo­phil­ia A, in­vestors quick­ly clipped 33% off of Spark’s share price this morn­ing at the un­steady re­sults it pro­duced.

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