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Company Spark Therapeutics Inc.
DescriptionAdeno-associated virus (AAV) that delivers the Factor VIII gene
Molecular Target Factor VIII
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationHemophilia
Indication DetailsTreat hemophilia A
Regulatory Designation

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