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Company Sangamo BioSciences Inc.
DescriptionAdeno-associated virus (AAV) vector containing zinc finger DNA binding protein (ZFP) nucleases (ZFNs) targeting the albumin locus and the gene for alpha-L-iduronidase (IDUA)
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy
Latest Stage of DevelopmentPhase I
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis I (MPS I, Hurler syndrome)
Regulatory Designation U.S. - Orphan Drug (Treat mucopolysaccharidosis I (MPS I, Hurler syndrome))

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