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 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

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

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.