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Company Spark Therapeutics Inc.
DescriptionAdeno-associated viral serotype 8 (AAV8) vector encoding human Factor IX
Molecular Target Factor IX
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationHemophilia
Indication DetailsTreat hemophilia B
Regulatory Designation

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