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Company RegenxBio Inc.
DescriptionAdeno-associated virus serotype 9 (AAV9) vector that delivers the human alpha-L-iduronidase (IDUA) gene to the CNS
Molecular Target Alpha-L-iduronidase (IDUA)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis I (MPS I, Hurler syndrome)
Regulatory Designation U.S. - Orphan Drug (Treat mucopolysaccharidosis I (MPS I, Hurler syndrome));
U.S. - Rare Pediatric Disease (Treat mucopolysaccharidosis I (MPS I, Hurler syndrome))

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