Rebisufligene etisparvovec is an AAV9-based SGSH gene therapy indicated for neurologic manifestations of mucopolysaccharidosis type IIIA in pediatric patients with preserved neurodevelopmental function. Rebisufligene etisparvovec is an adeno-associated virus serotype 9 (AAV9)-based gene therapy that delivers a functional copy of the human SGSH gene, leading to production of the lysosomal enzyme sulfamidase.
- Mechanism
- Curator reviewed · 3 references
Rebisufligene etisparvovec is an AAV9-based gene therapy designed to deliver a functional copy of the human N-sulfoglucosamine sulfohydrolase (SGSH) gene to cells. The vector is a non-replicating, recombinant AAV9 containing the cDNA of the R169Q variant of the human SGSH gene under the control of the murine small nuclear RNA promoter U1a. Intravenous infusion results in production of the lysosomal enzyme sulfamidase in target tissues, with subsequent breakdown of heparan sulfate in lysosomes.
- Primary indication
- Rebisufligene etisparvovec is indicated for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function.Curator reviewed · 1 structured indication
- Also known as
- scAAV9.U1a.hSGSH · UX111
- Code names
- ABO-102
Resolves to
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