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BMN 270

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Company BioMarin Pharmaceutical Inc.
DescriptionAdeno-associated viral (AAV) vector delivering the Factor VIII gene
Molecular Target Factor VIII
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationHemophilia
Indication DetailsTreat hemophilia A
Regulatory Designation U.S. - Orphan Drug (Treat hemophilia A)
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