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zafinofusp alfa
- Development stage
- Unknown
- Lead developer
- Denali Therapeutics
- Modality
- Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Zafinofusp alfa (DNL126) is an investigational, intravenously administered, Enzyme Transport Vehicle (ETV)-enabled N-sulfoglucosamine sulfohydrolase (SGSH) replacement therapy developed by Denali Therapeutics. It is designed to cross the blood-brain barrier (BBB) via receptor-mediated transcytosis by binding to the transferrin receptor, enabling the delivery of the functional SGSH enzyme into the brain and peripheral tissues. Zafinofusp alfa is being developed for the treatment of Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA, MPS IIIA), a rare lysosomal storage disorder caused by a deficiency in the SGSH enzyme, which leads to the accumulation of heparan sulfate. The drug is currently in Phase 1/2 clinical development.
- Other names
- zafinofusp alfa
02
Targets
TFRC (Transferrin Receptor)HS (Heparan sulfate)
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