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Company uniQure N.V.
DescriptionAdeno-associated viral serotype 5 (AAV5) vector carrying the N-acetylglucosaminidase alpha (NAGLU) gene
Molecular Target N-acetylglucosaminidase alpha (NAGLU)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo type B syndrome)
Regulatory Designation

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