Spark Therapeutics (Nasdaq: ONCE) was down 28% on Tuesday lunchtime following a serious adverse event during a Phase I/II trial of its experimental hemophilia A genetic therapy.
A couple of patients are reported to have suffered alarming immune system responses to SPK-8011, with one requiring intravenous infusions in hospital. In total, seven of the 12 trial participants needed to be administered steroids to cope with side-effects of SPK-8011.
It's this news that led to Spark stock cratering and that of BioMarin (Nasdaq: BMRN) — which is currently in the race with Spark to get the first hemophilia gene therapy approval — to rise by around 7%. Spark, however, has resolved to push on to Phase III.
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