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ZFN Therapeutic for hemophilia B (SB-FIX)

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Company Sangamo BioSciences Inc.
DescriptionZinc finger DNA-binding protein nuclease (ZFN) targeting mutated Factor IX
Molecular Target Factor IX
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy
Latest Stage of DevelopmentPhase I
Standard IndicationHemophilia
Indication DetailsTreat hemophilia B
Regulatory Designation U.S. - Orphan Drug (Treat hemophilia B)
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