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LYS-SAF-302

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Company Lysogene S.A.
DescriptionSurgically administered adeno-associated virus (AAV) serotype 10 (AAV10) vector carrying human N-sulfoglucosamine sulfohydrolase (SGSH; HNS)
Molecular Target
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 A syndrome)
Regulatory Designation U.S. - Orphan Drug (Treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo A syndrome));
U.S. - Rare Pediatric Disease (Treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo A syndrome))
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