JCR Pharmaceuticals
Together We Soar.
JCR is pleased to highlight our clinical achievements with nine presentations at WORLDSymposium 2021.
Download an overview of our WORLDSymposium abstracts Learn More About WORLDSymposium
JCR Pharmaceuticals Co., Ltd. is a global specialty pharmaceuticals company that is redefining expectations and expanding possibilities for people with rare and genetic diseases worldwide. Our core values – reliability, confidence, and persistence – means that the work we do benefits all our stakeholders, including employees, partners, and patients.
We continue to build upon our 45-year legacy in Japan while expanding our global footprint with trials in the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies.
Patient Commitment
We Stand with Patients
At JCR, we are united with the patients, families, and communities we serve. We seek to understand their needs and develop new medicines with significant and meaningful benefits. We created JCR to improve the lives of people with rare and genetic diseases, and we remain driven by this common purpose.
We recognize the limitations of available therapies for rare and genetic diseases, including lysosomal storage disorders such as MPS Type I, MPS Type II, and MPS Type III. Many people with these conditions experience severe complications of the central nervous system (CNS). Developmental delays, impaired cognition, and an overall poor prognosis can negatively impact independence and quality of life for both patients and caregivers.
Together, we are working towards better outcomes for patients.
Global Expansion
We continue to build upon our history of success in Japan while expanding our global footprint by developing first-in-class and best-in-class therapies, and further expanding our capabilities in manufacturing and R&D.
Trials Beginning in 2021
The United States
Europe
Latin America
Recent News Milestones
| September 2020 |
Received Orphan Drug Designation for JR-141 (MPS II) in Japan Filed for marketing approval of JR-141 (MPS II) in Japan |
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| October 2020 | First patient dosed in phase 1/2 of global clinical trial of JR-171 (MPS I) |
| December 2020 | Filed for marketing authorization of JR-141 (MPS II) in Brazil |
View our WORLDSymposium 2021 Abstracts
This year, JCR Pharmaceuticals is attending the virtual 17th annual WORLDSymposium, an annual conference dedicated to lysosomal storage disorders, Feb. 8-12. We will have nine presentations at the event.
Development Pipeline – January 2021
| Target Indicators | Basic Research | Animal Model Study |
Process Development* Including development of industrial scale process, testing method and formulation | Preclinical | Clinical (Global) | Application for marketing approval |
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Target Indicator: MPS II - Hunter JR-141 (Japan and Brazil) Development Stage: Application for marketing approval |
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Target Indicator: MPS II - Hunter JR-141 (U.S., EU, and Latin America) Development Stage: Clinical (Global) |
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Target Indicator: MPS II - Hurler JR-171 Development Stage: Clinical (Global) |
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Target Indicator: Pompe disease JR-162 Development Stage: Preclinical |
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Target Indicator: MPS IIIA - Sanfilippo A JR-441 Development Stage: Preclinical |
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Target Indicator: MPS VII - Sly JR-443 Development Stage: Process Development* Including development of industrial scale process, testing method and formulation |
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Target Indicator: MPS IIB - Sanfilippo B JR-446 Development Stage: Process Development* Including development of industrial scale process, testing method and formulation |
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Target Indicator: GM1 gangliosidosis Development Stage: Animal Model Study |
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Target Indicator: Fucosidosis Development Stage: Animal Model Study |
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Target Indicator: GM2 gangliosidosis - Tay-Sachs disease Development Stage: Animal Model Study |
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Target Indicator: Krabbe disease Development Stage: Animal Model Study |
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Target Indicator: Metachromatic leukodystrophy Development Stage: Animal Model Study |
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Target Indicator: CLN1 - Batten disease infantile Development Stage: Animal Model Study |
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Target Indicator: CLN2 - Batten disease late infantile Development Stage: Animal Model Study |
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Target Indicator: Gaucher disease Development Stage: Animal Model Study |
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Target Indicator: Niemann-Pick disease Development Stage: Animal Model Study |
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Target Indicator: ɑ-Mannosidosis Development Stage: Animal Model Study |
J-Brain Cargo® Technology
Our first-in-class proprietary technology, J-Brain Cargo®, enables us to develop therapies that cross the blood-brain barrier and penetrate the central nervous system (CNS). The CNS complications of diseases are often severe, resulting in developmental delays, an impact on cognition and, above all, poor prognosis, which affect patients’ independence as well as the quality of life of patients and their caregivers. With J-Brain Cargo®, we seek to address the unresolved clinical challenges of lysosomal storage disorders by delivering the enzyme to both the body and the brain.