Spark Therapeutics’ SPK-8011 is an adeno-associated virus-mediated gene therapy, aiming to restore normal clotting function to patients with hemophilia A. SPK-8011 delivers B domain-deleted coagulation factor VIII (fVIII), and encodes the same amino acid sequence as found in the widely used recombinant product Xyntha (Pfizer). SPK-8011 is currently midway through a Phase II trial that the company is wholly funding, unlike its gene therapy candidate for hemophilia B, SPK-9001, which is partnered to Pfizer for development and commercialization.
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