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Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here.


Spark Therapeutics and Pfizer Inc. announced today that the聽U.S. Food and Drug Administration聽(FDA) has granted breakthrough therapy designation to聽SPK-9001,聽the lead investigational candidate in the companies’聽SPK-FIX聽program, in development for the treatment of hemophilia B.聽SPK-9001,聽a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized, high-activity human factor IX variant, is being investigated in an ongoing Phase 1/2 trial as a potential one-time therapy.

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