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Company RegenxBio Inc.
DescriptionAdeno-associated virus serotype 9 (AAV9) vector that delivers the iduronate 2-sulfatase (IDS) gene
Molecular Target Iduronate 2-sulfatase (IDS)
Mechanism of Action 
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis II (MPS II, Hunter syndrome)
Regulatory Designation U.S. - Rare Pediatric Disease (Treat mucopolysaccharidosis II (MPS II, Hunter syndrome))

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