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24.12.2025
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Italfarmaco and JCR Pharmaceuticals Announce Commercialisation Agreement for Givinostat in Japan and Strategic Collaboration in Rare Disease
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Italfarmaco S.p.A. and JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), today announced an exclusive licensing agreement for the development and commercialisation of givinostat in Japan.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251223789271/en/
Under the terms of the agreement, J...
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07.10.2025
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JCR Pharmaceuticals Enhances Delivery of AAV Gene Therapy to Central Nervous System and Muscle with Novel Capsid Engineering Platform Technology
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today presented non-clinical data demonstrating the ability of its proprietary JUST-AAV capsid engineering platform to achieve efficient delivery of adeno-associated virus (AAV) gene therapy...
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01.10.2025
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JCR Pharmaceuticals Announces Licensing Agreement with Menagen to Commercialize Agalsidase Beta BS I.V. Infusion [JCR] Across Nine MENAT Markets
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced an exclusive licensing agreement with Menagen Pharmaceutical Industries LLC (headquartered in Oman, CEO: Saif Al Hasani, “Menagen”) to seek local marketing authorizations and...
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20.10.2025
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30.09.2025
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JCR Pharmaceuticals to Present at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it will present non-clinical data from its novel JUST-AAV gene therapy platform technology in an oral session at the European Society of Gene and Cell Therapy (ESGCT) 32...
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29.09.2025
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Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by Ministry of Health, Labour and Welfare of Japan
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MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the Ministry of Health, Labour and Welfare of Japan has granted orphan drug designation to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B).
This press...
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19.09.2025
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JCR Pharmaceuticals and Alexion Achieve Milestone in Collaborative Neurodegenerative Disease Program
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, has achieved a key research milestone in collaboration with Alexion, AstraZeneca Rare Disease (“Alexion”), focused on applying JCR’s proprietary J-Brain Cargo® technology to develop a therap...
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12.09.2025
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JCR Pharmaceuticals Celebrates its 50th Anniversary of Advancing Science and Changing Lives
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced that it will celebrate its 50th anniversary on September 13, 2025, marking five decades of advancing science and redefining possibilities for patients living with complex hea...
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08.09.2025
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JCR Pharmaceuticals Presents Long-Term Clinical Data on Pabinafusp Alfa for the Treatment of Mucopolysaccharidosis Type II (MPS II) at ICIEM 2025
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, presented new data from its pipeline of brain-penetrant enzyme replacement therapies at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, held September 2-6, 2025...
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02.09.2025
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MEDIPAL HOLDINGS and JCR Pharmaceuticals Sign Global Licensing and Japan Co-Development Agreement for JR-479, a Novel Therapy for GM2 Gangliosidosis
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MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) announced that the two companies have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis, an ultra-rare lysosomal storage disorder....
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28.08.2025
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JCR Pharmaceuticals to Present at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) 2025
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it will present data at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) 2025, held September 2-6, 2025, in Kyoto, Japan. The presentations will de...
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16.07.2025
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JCR Pharmaceuticals Selected for Japan’s Regenerative Medicine CDMO Subsidy Program
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, has been selected for the Ministry of Economy, Trade and Industry’s “Regenerative CDMO Subsidy” as of July 15, 2025. The subsidy will support facility upgrades and equipment installation to...
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15.07.2025
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JCR Pharmaceuticals and Acumen Pharmaceuticals Announce Collaboration to Develop Therapy for Alzheimer’s Disease, Enabled by J-Brain Cargo® Technology Platform
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; JCR), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced a joint collaboration, option and license agreement with Acumen Pharmaceuticals to develop a novel therapeutic candidate for the treatment of Alzheimer’s disease (AD), enabled...
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08.07.2025
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JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) today announced that it has entered into a license agreement with Alexion, AstraZeneca Rare Disease (“Alexion”), for JCR’s new, proprietary JUST-AAV capsids to develop genomic medicines.
JUST-AAV encompasses a range of vector types optimized for various target tissues—including liver-sparing, muscl...
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02.07.2025
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JCR Pharmaceuticals Announces the Achievement of Enrollment in the JR-141 Global Phase III Clinical Trial
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; JCR) announced that it achieved the enrollment of the target number of participants in the global Phase III clinical trial of JR-141 (INN: pabinafusp alfa), which is in development for the treatment of mucopolysaccharidosis type II (MPS II, also known as Hunter syndrome). The Phase III clinical trial is ong...
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24.06.2025
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Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European Commission (EC)
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MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the European Commission (EC) has granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B), following the recent the U.S...
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18.06.2025
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JCR Pharmaceuticals Announces Launch of Mucopolysaccharidosis (MPS) Type II Disease Awareness Film Featured in The Next Frontier Series
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced the launch of a film featured in The Next Frontier, a global branded film series presented by the Biotechnology Innovation Organization (BIO) and produced by BBC StoryWorks Commerc...
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06.06.2025
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JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced the launch of its new global website. As the company turns 50, it is marking its growing footprint in Japan, the U.S., Europe, and Latin America with a unique website tailore...
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16.05.2025
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JCR Pharmaceuticals Presents Preclinical Gene Therapy Data that Demonstrate Promising CNS Uptake at American Society of Gene and Cell Therapy 28th Annual Meeting
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, being held May 13-17, 2025, in New Orleans, LA. In an oral presentation, the JCR researc...
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08.05.2025
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JCR Pharmaceuticals to Present at the American Society of Gene and Cell Therapy 28th Annual Meeting
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that it will present updated preclinical data from its proprietary J-Brain Cargo®-applied adeno-associated virus (AAV) vector gene therapy non-clinical research programs in an oral abstract session at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, being hel...
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07.05.2025
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Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA
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MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B).
This press releas...
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05.02.2025
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JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2025 Showcase Research from Its Investigational Treatments for Lysosomal Storage Disorders
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) today announced the presentation of two datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs) at the 21st Annual WORLDSymposiumTM 2025. JCR is presenting new data from a pair of its programs that apply its J-Brain Cargo® platform, a prop...
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28.01.2025
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JCR Pharmaceuticals to Present at the 21st Annual WORLDSymposium™ 2025
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that it will present at the 21st Annual WORLDSymposium™ 2025, held February 3-7, 2025, in San Diego, Calif. The poster presentations will demonstrate the potential benefits of the investigational therapies in JCR’s development pipeline and of J-Brain Cargo®, JCR’s proprietary technolo...
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06.01.2025
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JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy
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JCR Pharmaceuticals Co., Ltd. (TOKYO:4552; “JCR”) and Modalis Therapeutics Corporation (TOKYO:4883; “Modalis”) today announced that they have validated the initial proof of concept in a joint research program for the development of a novel gene therapy for a central nervous system (CNS) disease. Due to the success of the partnership thus far, Moda...
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20.12.2024
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JCR Pharmaceuticals Initiates Phase III Clinical Trial of JR-142 in Japan, a Long-Acting Growth Hormone Therapy
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) today announced that the first patient has been dosed in the Phase III clinical trial of JR-142 (INN: redalsomatropin alfa) in Japan, marking a significant milestone in the development of this innovative treatment. JR-142 is a long-acting growth hormone therapy and is being studied in patients with p...
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05.12.2024
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MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Announce Initiation of First Dosing in Phase I/II Clinical Trial of JR-446 for Mucopolysaccharidosis Type IIIB in Japan
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MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced the initiation of the Phase I/II clinical trial of JR-446 in Japan following the dosing of the first individual from the trial. JR-446 is a proprietary blood-brain barrier (BBB)-penetrating α-N-acetylglucosaminidase in development for...
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31.10.2024
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JCR Pharmaceuticals Initiates First Dosing in Phase I Clinical Trial of JR-441 for Mucopolysaccharidosis Type IIIA
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced the initiation of the first patient dosing in Japan in the Phase I clinical trial of JR-441, an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). JR-441 is a proprietary recombinant heparan N...
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23.10.2024
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JCR Pharmaceuticals Presents Preclinical Gene Therapy Research at the European Society of Gene and Cell Therapy 31st Annual Congress
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs in a poster session at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, being held October 22-25, 2024...
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16.10.2024
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JCR Pharmaceuticals to Present at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present preclinical data from its proprietary J-Brain Cargo®-applied adeno-associated virus (AAV) gene therapy preclinical research programs in a poster session at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congr...
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05.09.2024
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JCR Pharmaceuticals’ Data Presentations at SSIEM Annual Symposium 2024 Highlight Investigational Treatments for Lysosomal Storage Disorders
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JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held in Porto, Portugal from September 3-6, 2024. We highlighted our proprietary J-Brain Cargo® technology through two key presentations focused on advanced therapies for lysosomal stor...
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03.09.2024
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JCR Pharmaceuticals Announces Completion of Clinical Trial Notification Process in Japan for Phase I Study of JR-441 for Mucopolysaccharidosis Type IIIA
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JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today the completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for the clinical trial notification for the Phase I study of JR-441 in individuals with mucopolysaccharidosis type IIIA (MPS IIIA; Sanfilippo syndrome...
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