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JR-141 (pabinafusp alfa)

Hunter Syndrome (MPS II)

ApprovedMarketed in Japan/Brazil; Regulatory review elsewhere

Key Facts

Indication

Hunter Syndrome (MPS II)

Phase

Approved

Status

Marketed in Japan/Brazil; Regulatory review elsewhere

Company

JCR Pharmaceuticals

About JCR Pharmaceuticals

JCR Pharmaceuticals has established itself as a premier global specialty pharmaceutical company focused on high-unmet-need rare diseases, particularly lysosomal storage disorders. Its strategic advantage is anchored in the proprietary J-Brain Cargo® platform, which enables targeted delivery of biologics across the blood-brain barrier—a key differentiator in treating neurological manifestations. The company has successfully commercialized products in multiple international markets and maintains a robust clinical pipeline, driving its significant market valuation. JCR's fully integrated model, from R&D to global commercialization, combined with a strong venture spirit, positions it for sustained growth in the orphan drug space.

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Therapeutic Areas

Lysosomal Storage Disorders Neurological Diseases Rare Genetic Diseases

Other Hunter Syndrome (MPS II) Drugs

Drug	Company	Phase	Status
MPS II (Hunter Syndrome) Program	NeuroGT	Pre-clinical	Active
HMI-106	Homology Medicines	Preclinical	Discontinued
RG6356 (DNL310)	Roche	Phase II/III	Active
DNL310	Denali Therapeutics	Phase 2/3	Active