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heparan-N-sulfatase (human, recombinant)
- Development stage
- Unknown
- Lead developer
- Takeda
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intrathecal, Intravenous
01
Overview
Recombinant human heparan-N-sulfatase (rhHNS) is an investigational enzyme replacement therapy developed for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A), a lysosomal storage disorder characterized by deficiency of the enzyme sulfamidase. rhHNS is a recombinant protein designed to replace the deficient endogenous enzyme and degrade accumulated heparan sulfate in affected tissues. Because intravenously administered enzymes do not cross the blood-brain barrier, rhHNS has been formulated for intrathecal administration via a surgically implanted drug delivery device to target central nervous system involvement in MPS IIIA. Clinical studies have shown that intrathecal administration results in appreciable CNS tissue distribution; however, long-term studies indicate that while generally well-tolerated, rhHNS did not slow neurocognitive decline in treated patients[2][3][4][5][6].
- Other names
- recombinant human heparan-N-sulfatase
02
Targets
HS (Heparan sulfate)
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