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JCR Pharmaceuticals Co., Ltd.

JCR Pharmaceuticals Co., Ltd.

  • Who We Are
  • Patient Commitment
  • Global Expansion
  • Pipeline
  • J-Brain Cargo Technology
  • Contact

Partnering with JCR
Together We Soar.

JCR is pleased to highlight our clinical achievements with four oral presentations and 10 posters at WORLDSymposium™ 2023.

Download an overview of our WORLDSymposium™ 2023 abstracts Learn More About WORLDSymposium™

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.

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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 48-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

Doctor checking child's heartbeat
Doctor checking child's heartbeat

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
  • Launched 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 (MPS III A - Sanfilippo A) from European Commission
February 2022
  • Dosed first patient in phase 3 of global clinical trial of JR-141
  • IZCARGO® received the 2022 WORLDSymposium™ New Treatment Award
March 2022
  • Entered development phase of JR-479 (GM2 gangliosidosis - Tay-Sachs disease)
  • Concluded exclusive license and collaborative agreement with Takeda to develop gene therapies using J-Bran Cargo®
  • Technology for LSDs
July 2022
  • Entered into and executed agreement for an underwriting of a third-party allotment of new shares from Mycenax Biotech Inc., a CDMO in Taiwan
September 2022
  • Established JCR Europe B.V. as a subsidiary to engage in development activities in Europe
October 2022
  • Entered development phase for JR-471 (fucosidosis)
  • Concluded a licensing contract for commercialization of a Fucosidosis therapeutic with MEDIPAL HOLDINGS
  • MEDIPAL HOLDINGS and JCR Pharmaceuticals agree on the granting of exclusive negotiation rights for global commercialization targeting four ultra-rare diseases and conclude a licensing contract for the commercialization of a fucosidosis therapeutic
January 2023
  • Granted Rare Pediatric Disease Designation for JR-141 for the Treatment of MPS II by US FDA
March 2023
  • JCR Pharmaceuticals announces research collaboration, option and license agreement with Alexion to develop a therapy using J-Brain Cargo® for neurodegenerative disease
May 2023
  • Angelini Pharma and JCR Pharmaceuticals announce global collaboration for the development and commercialization of novel biologic therapies in epilepsy
July 2023
  • Received approval to start global phase I/II clinical trial for JR-441 for the treatment of MPS Type III A by the PEI (Paul-Ehrlich Institute)

Learn More about our recent news milestones

View our WORLDSymposium™ 2023 Abstracts

This year, JCR Pharmaceuticals attended the virtual 19th annual WORLDSymposium™, an annual conference dedicated to lysosomal storage disorders, Feb. 22-26. We had four oral presentations and 10 posters at the event.

Download an overview of our WORLDSymposium™ abstracts

Development Pipeline – October 2023

Target Indicators Basic Research Animal
Model Study
Process Development* Including development of industrial scale process, testing method and formulation Preclinical Clinical 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 (U.S., EU, and Latin America)
Development Stage: Clinical (Global)
JR-141 (US, Europe, Asia, 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: GM2 gangliosidosis - Tay‑Sachs disease
Development Stage: Process Development* Including development of industrial scale process, testing method and formulation
JR-479
Target Indicator: Fucosidosis
Development Stage: Process Development* Including development of industrial scale process, testing method and formulation
JR-471
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
JR-194
Target Indicator: GM1 gangliosidosis
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

*Including development of industrial scale process, testing method and formulation

**Approved in Japan march 2021 and launched in May 2021

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.

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Please visit jcrpharm.co.jp for more information.