Partnering with JCR
Together We Soar.

We will continuously try to create value by our unique technology platform such as J-Brain Cargo® toward our corporate philosophy: “contributing towards people’s healthcare through pharmaceutical products.”

In addition to the in-house research activities, we think collaborative research and development work with other companies are also important. The values of our technology platform will be increased by integrating our technologies and other technologies and by implementing our technology in other companies’ products. We aim at “research-oriented specialty pharma with global exposure” utilizing collaborative research and development work.

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 belief – means that the work we do benefits all our stakeholders, including employees, partners, and patients.

We continue to build upon our 46-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. If you are interested in speaking with JCR regarding business development opportunities, email bd@jcrpharm.co.jp.

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.

Read a message from our CEO

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.

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
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
February 2021	Cleared FDA IND for JR-141 Received Fast Track Designation for JR-141 (MPS II) from FDA Received Orphan Drug Designation for JR-171 (MPS I) from FDA
March 2021	Obtained marketing approval of JR-141 (MPS II) from MHLW in Japan Received Orphan Drug Designation for JR-171 from European Commission
May 2021	Launch of pabinafusp alfa (MPS II) in Japan
September 2021	Received Fast Track Designation for JR-171 (MPS I) from FDA Conclusion of an exclusive co-development and commercialization agreement for JR-141 with Takeda Pharmaceutical Co., Ltd in certain regions JR-171 was granted Fast track designation
October 2021	Received PRIME Designation for JR-141 (MPS II) from EMA
January 2022	Received Orphan Drug Designation for JR-441 from FDA

Learn More about our recent news milestones

View our WORLDSymposium 2022 Abstracts

This year, JCR Pharmaceuticals is attending the virtual 18th annual WORLDSymposium, an annual conference dedicated to lysosomal storage disorders, Feb. 7-11. We will have six presentations at the event.

Download an overview of our WORLDSymposium abstracts

Development Pipeline – January 2022

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	Approved/Launch
Target Indicator: MPS II - Hunter JR-141 (Japan) Development Stage:** Approved/Launch	JR-141 (Japan)
Target Indicator: MPS II - Hunter JR-141 (Brazil) Development Stage:** Application for marketing approval	JR-141 (Brazil)
Target Indicator: MPS II - Hunter JR-141 (U.S., EU, and Latin America) Development Stage: Clinical (Global)	JR-141 (U.S., EU, and Latin America)
Target Indicator: MPS I - Hurler etc. JR-171 Development Stage: Clinical (Global)	JR-171
Target Indicator: Pompe disease JR-162 Development Stage: Preclinical	JR-162
Target Indicator: MPS IIIA - Sanfilippo A JR-441 Development Stage: Preclinical	JR-441
Target Indicator: MPS IIB - Sanfilippo B JR-446 Development Stage: Preclinical	JR-446
Target Indicator: MPS VII - Sly JR-443 Development Stage: Preclinical	JR-443
Target Indicator: GM1 gangliosidosis Development Stage: Process Development
Target Indicator: Fucosidosis Development Stage: Process Development* Including development of industrial scale process, testing method and formulation
Target Indicator: GM2 gangliosidosis - Tay-Sachs disease Development Stage: Process Development* Including development of industrial scale process, testing method and formulation
Target Indicator: Krabbe disease Development Stage: Process Development* Including development of industrial scale process, testing method and formulation
Target Indicator: CLN1 - Batten disease infantile Development Stage: Animal Model Study
Target Indicator: Metachromatic leukodustrophy Development Stage: Animal Model Study
Target Indicator: CLN2 - Batten disease late infantile Development Stage: Process Development* Including development of industrial scale process, testing method and formulation
Target Indicator: Gaucher disease Development Stage: Animal Model Study
Target Indicator: Niemann-Pick disease Development Stage: Animal Model Study
Target Indicator: ɑ-Mannosidosis Development Stage: Animal Model Study
Target Indicator: Galactosialidosis Development Stage: Basic Research

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.

Discover More About our R&D Capabilities

Contact

If you are interested in speaking with JCR regarding business development opportunities, email bd@jcrpharm.co.jp.

Partnering with JCR Together We Soar.