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adenovirus-associated viral vector serotype 9 carrying the human sulfamidase gene, Sanfilippo

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Company Esteve S.A.
DescriptionAdenovirus-associated viral (AAV) vector serotype 9 (AAV9) carrying the human N-sulfoglucosamine sulfohydrolase (SGSH; HNS) gene
Molecular Target N-sulfoglucosamine sulfohydrolase (SGSH) (HNS)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPreclinical
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome)
Regulatory Designation

EU - Orphan Drug (Treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome))

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