SPK-9001 is Spark Therapeutics’ leading candidate in development for hemophilia B. Like SPK-8011, SPK-9001 is also an adeno-associated virus-mediated gene therapy, but instead delivers factor IX (fIX). Specifically, SPK-9001 encodes the Padua variant of fIX, which has eightfold greater clotting activity than the normal protein.
In December 2014, Pfizer signed an exclusive deal with Spark Therapeutics to collaborate on the development of SPK-9001. Pfizer will assume full clinical responsibility and commercialization rights should the drug progress to Phase III, which is expected to occur during 2018.
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