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JR-141
JR-141 is a recombinant human arylsulfatase A enzyme designed to cross the blood-brain barrier and replace deficient enzyme activity in lysosomal storage disorders.
JR-141 is a recombinant human arylsulfatase A enzyme designed to cross the blood-brain barrier and replace deficient enzyme activity in lysosomal storage disorders. Used for Metachromatic leukodystrophy (MLD).
At a glance
| Generic name | JR-141 |
|---|---|
| Sponsor | JCR Pharmaceuticals Co., Ltd. |
| Drug class | Enzyme replacement therapy |
| Target | Arylsulfatase A |
| Modality | Small molecule |
| Therapeutic area | Rare genetic/lysosomal storage disorders |
| Phase | Phase 3 |
Mechanism of action
JR-141 uses a blood-brain barrier penetrating peptide technology to deliver functional arylsulfatase A enzyme into the central nervous system. This enzyme replacement therapy aims to restore enzymatic activity and reduce accumulation of sulfatide substrates in the brain and other tissues affected by arylsulfatase A deficiency. The drug is being developed for metachromatic leukodystrophy (MLD), a rare lysosomal storage disease.
Approved indications
- Metachromatic leukodystrophy (MLD)
Common side effects
- Infusion-related reactions
- Immune responses/antibody formation
Key clinical trials
- A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) (PHASE3)
- An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects (PHASE3)
- A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21) (PHASE2)
- An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II (PHASE2, PHASE3)
- An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II (PHASE2)
- A Study of JR-141 in Patients With Mucopolysaccharidosis II (PHASE2, PHASE3)
- A Study of JR-141 in Patients With Mucopolysaccharidosis Type II (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- JR-141 CI brief — competitive landscape report
- JR-141 updates RSS · CI watch RSS
- JCR Pharmaceuticals Co., Ltd. portfolio CI