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lepunafusp 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
- Administration
- Intravenous
01
Overview
Lepunafusp alfa is a recombinant fusion protein designed as an enzyme replacement therapy for mucopolysaccharidosis type I (MPS I). It consists of human α-L-iduronidase (IDUA) fused to a humanized anti-human transferrin receptor antibody fragment, enabling the therapeutic enzyme to cross the blood-brain barrier via transferrin receptor-mediated transcytosis. This design allows for both systemic and central nervous system delivery, addressing somatic and neurocognitive symptoms of MPS I. Lepunafusp alfa was developed using JCR Pharmaceuticals' proprietary J-Brain Cargo technology and has received orphan drug designation for MPS I[1][4][5][6].
- Other names
- lepunafusp alfaJR-171JR171JR 171
02
Targets
TFRC (Transferrin Receptor)HS/DS (Heparan sulfate and dermatan sulfate)
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