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Company Sangamo BioSciences Inc.
DescriptionZFN-mediated genome editing that runs on the In Vivo Protein Replacement Platform (IVPRP)
Molecular Target
Mechanism of Action 
Therapeutic Modality 
Latest Stage of DevelopmentPhase I
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis II (MPS II, Hunter syndrome)
Regulatory Designation

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