Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here.
Spark Therapeutics and Pfizer Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program, in development for the treatment of hemophilia B. SPK-9001, 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.
