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posnafusp alfa
- Development stage
- Phase 2
- Lead developer
- JCR Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes, Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
Posnafusp alfa is an investigational enzyme replacement therapy developed by JCR Pharmaceuticals Co., Ltd. for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. It is a recombinant fusion protein that combines heparan N-sulfatase, the enzyme deficient in MPS IIIA, with an antibody fragment targeting the human transferrin receptor. This design utilizes JCR's proprietary J-Brain Cargo® technology to enable the drug to cross the blood-brain barrier, addressing both somatic and neurological symptoms of the disease. It is administered via intravenous infusion.
- Other names
- n-sulfoglucosamine sulfohydrolase fused to a humanised monoclonal antibody targeting human transferrin receptor
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)TFRC (Transferrin Receptor)
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