Spark Plots Rebound For Hemophilia A Gene Therapy, As Rival BioMarin Surges

 
• By Emily Hayes

Levels of factor VIII in hemophilia A patients after treatment with new gene therapies are variable, a source of investor concern.

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Spark Therapeutics Inc. went into the American Society of Hematology annual meeting with its hemophilia A gene therapy SPK-8011 looking well matched against close competitor valoctocogene roxaparvovec (BMN 270) from BioMarin Pharmaceutical Inc., but was soon flattened by data that raised questions about consistency of response and commercialization timelines.

Spark's SPK-8011 (Spark200) has a novel bio-engineered adeno-associated viral (AAV) vector that encapsulates the human factor VIII gene under the control of a

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