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SAF-301

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Company Lysogene S.A.S.
DescriptionIntracerebrally administered adeno-associated virus (AAV) serotype 10 (AAV10) vector encoding N-sulfoglucosamine sulfohydrolase (SGSH; HNS) and sulfatase modifying factor 1 (SUMF1)
Molecular Target N-sulfoglucosamine sulfohydrolase (SGSH) (HNS) ; Sulfatase modifying factor 1 (SUMF1)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome)
Regulatory Designation

Partner

ReGenX Biosciences LLC


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