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Note: The following is an edited version of a press release from Spark Therapeutics.聽Read the press release from Spark Therapeutics聽in it鈥檚 entirety here.


The company has disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011,聽a novel bio-engineered adeno-associated viral (AAV) vector utilizing the Spark200 capsid and containing a codon-optimized human factor VIII gene, being investigated as a potential one-time therapy for hemophilia A. It is the second investigational hemophilia gene therapy to emerge from Spark Therapeutics’ leading gene therapy platform. Spark Therapeutics is developing SPK-8011 independently and retains global commercialization rights to the聽SPK-FVIII聽program.


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