[{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Announces Presentations on JR-141 and JR-162 at the 16th Annual WORLDSymposium 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"JAPAN","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Completes Phase 1\/2 Clinical Trial Notification of JR-171 for mucopolysaccharidosis type I","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Announces First Patient Dosed in Phase1\uff0f2 Global Clinical Trial of JR-171 for MPS I","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Fast Track Designation for JR-141 for the Treatment of Mucopolysaccharidosis Type II (Hunter Syndrome)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Receives FDA IND Clearance to Initiate Global P3 Trial of JR-141 for Mucopolysaccharidosis Type II (Hunter Syndrome)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Receives the WORLDSymposium\u2122 New Treatment Award for IZCARGO\u00ae (Pabinafusp Alfa)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals\u2019 Research Presentations at WORLDSymposium\u2122 2022 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Announces First Patient Dosed in Phase 3 Global Clinical Trial of JR-141 for Treatment of MPS II (Hunter Syndrome)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"JCR Pharmaceuticals Announces Research Collaboration, Option and License Agreement with Alexion to Develop a Therapy Using J-Brain Cargo\u24c7 for Neurodegenerative Disease","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Angelini Pharma","pharmaFlowCategory":"D","amount":"$505.5 million","upfrontCash":"Undisclosed","newsHeadline":"Angelini Pharma and JCR Pharmaceuticals Announce Global Collaboration for the Development and Commercialization of Novel Biologic Therapies in Epilepsy","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Sumitomo Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"JCR and Sumitomo Dainippon Pharma Enter into Marketing Alliance for Agalsidase Beta BS I.V. Infusion, for Treatment of Fabry Disease in Japan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"Biosimilar","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Announces 52-Week Interim Data from its Global Phase I\/II Study of JR-171 in Individuals with Mucopolysaccharidosis Type I (MPS I)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Announces First Patient Dosed in a Global Phase I\/II Clinical Trial of JR-441 for the Treatment of MPS IIIA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Modalis Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Modalis and JCR Pharmaceuticals Enter into Joint Research Agreement","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"JCR Pharmaceuticals Announces Achievement of Milestone Using J-Brain Cargo\u00ae Technology for Neurodegenerative Disease in Research Collaboration with Alexion","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"JCR Pharmaceuticals and Takeda Pharmaceuticals Announce Discontinuation of Gene Therapy Collaboration","therapeuticArea":"Technology","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Technology","graph2":"Discovery"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Grants Orphan Drug Designation to JR-441 for the Treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by JCR Pharmaceuticals
The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease.
Under the termination, Takeda will discontinue their collaboration with JCR to develop gene therapies using adeno-associated viruses (AAV) combined with the JCR J-Brain Cargo® Technology.
JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with Mucopolysaccharidosis Type IIIA (MPS IIIA).
Modalis and JCR Pharmaceuticals aim to establish new gene therapies for undisclosed CNS diseases by applying J-Brain Cargo®, JCR Pharmaceuticals’ proprietary technology for crossing the blood-brain barrier, and CRISPR-GNDM®, Modalis proprietary epigenome modulation technology.
JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.
JR-171 (lepunafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in subjects with MPS I.
Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a neurodegenerative disease.
The agreement includes the marketing alliance for JR-051 (agalsidase beta BS), a biosimilar of Fabrazyme, a therapeutic enzyme, being sold by JCR for the treatment of Fabry disease.
JR-141 is the first-ever approved enzyme replacement therapy (ERT) in Japan that penetrates the BBB via intravenous administration for the treatment of MPS II. JR-141 is expected to treat not only the systemic symptoms but also the neuronopathic manifestations of MPS II.
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