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recombinant human heparan-N-sulfatase
- Development stage
- Phase 2
- Lead developer
- Takeda
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intrathecal
01
Overview
Recombinant human heparan-N-sulfatase (rhHNS) is a protein-based enzyme replacement therapy developed for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A), a rare lysosomal storage disorder caused by deficiency of the endogenous enzyme heparan N-sulfatase. The drug is produced using recombinant DNA technology and is designed to replace the missing or deficient enzyme in affected patients, thereby enabling degradation of accumulated glycosaminoglycan heparan sulfate and reducing disease symptoms. Clinical trials have investigated its safety and efficacy in pediatric patients with early-stage MPS IIIA[1][4][5][8].
- Other names
- Heparan-N-sulfatase (human, recombinant)
02
Targets
SGSH (N-sulphoglucosamine sulphohydrolase)
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