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JR-471
- Development stage
- Preclinical
- Lead developer
- JCR Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
JR-471 is a recombinant enzyme replacement therapy (ERT) being developed by JCR Pharmaceuticals for the treatment of fucosidosis, a rare and progressive lysosomal storage disorder. The drug consists of a recombinant form of the enzyme α-L-fucosidase, which is deficient in affected individuals. JR-471 incorporates JCR's proprietary J-Brain Cargo® platform technology, designed to transport the therapeutic enzyme across the blood-brain barrier (BBB) into the central nervous system via receptor-mediated transcytosis. This approach aims to address both the systemic and neurological manifestations of the disease. As of the current reports, JR-471 is in the preclinical stage of development.
- Other names
- recombinant alpha-L-fucosidaseBBB-penetrating alpha-L-fucosidase
02
Targets
TFRC (Transferrin Receptor)Terminal alpha-L-fucose residues on glycoconjugates
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