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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
JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines

Associated Press

time08-07-2025

  • Business
  • Associated Press

JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines

HYOGO, Japan--(BUSINESS WIRE)--Jul 8, 2025-- 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, muscle-targeting, and brain-targeting variants—to expand the potential of AAV-based gene therapy. Under the terms of the agreement, Alexion may use the licensed capsids, which are part of the JUST-AAV platform, in up to five of Alexion's genomic medicines programs. JCR will receive an upfront payment from Alexion. JCR is eligible to receive milestone payments of up to USD 225 million related to research and development, and up to USD 600 million related to sales, for a total of up to USD 825 million (approximately JPY 120 billion converted at the exchange rate of JPY 145 to USD), which reflects the aggregate milestones if all five targets are exercised. In addition, JCR is entitled to receive tiered royalties based on net sales. This agreement marks the third partnership between JCR and Alexion, following research collaborations involving J-Brain Cargo ® technology— the first announced in March 2023 for neurodegenerative disease and the second announced in December 2023 for the discovery of oligonucleotide therapeutics. 'I am very pleased to announce the signing of this license agreement for our proprietary AAV capsid platform, JUST-AAV,' said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. 'I believe JUST-AAV has the potential to make a real difference in the development of gene therapies for rare diseases, and I look forward to its application across a broader range of conditions. JCR values its partnership with Alexion, and we are pleased to build on the collaboration we have developed over the years.' The impact of this agreement on JCR's consolidated financial results for the fiscal year ending March 31, 2026, has already been factored into the current earnings forecast. View source version on CONTACT: Investors & Media: JCR Pharmaceuticals Co., Ltd. Corporate Communications [email protected] KEYWORD: JAPAN ASIA PACIFIC INDUSTRY KEYWORD: SCIENCE OTHER SCIENCE BIOTECHNOLOGY RESEARCH PHARMACEUTICAL GENERAL HEALTH HEALTH HEALTH TECHNOLOGY SOURCE: JCR Pharmaceuticals Co., Ltd. Copyright Business Wire 2025. PUB: 07/08/2025 01:58 PM/DISC: 07/08/2025 01:57 PM

JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines
JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines

Business Wire

time08-07-2025

  • Business
  • Business Wire

JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines

HYOGO, Japan--(BUSINESS WIRE)-- 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, muscle-targeting, and brain-targeting variants—to expand the potential of AAV-based gene therapy. Under the terms of the agreement, Alexion may use the licensed capsids, which are part of the JUST-AAV platform, in up to five of Alexion's genomic medicines programs. JCR will receive an upfront payment from Alexion. JCR is eligible to receive milestone payments of up to USD 225 million related to research and development, and up to USD 600 million related to sales, for a total of up to USD 825 million (approximately JPY 120 billion converted at the exchange rate of JPY 145 to USD), which reflects the aggregate milestones if all five targets are exercised. In addition, JCR is entitled to receive tiered royalties based on net sales. This agreement marks the third partnership between JCR and Alexion, following research collaborations involving J-Brain Cargo ® technology— the first announced in March 2023 for neurodegenerative disease and the second announced in December 2023 for the discovery of oligonucleotide therapeutics. 'I am very pleased to announce the signing of this license agreement for our proprietary AAV capsid platform, JUST-AAV,' said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. 'I believe JUST-AAV has the potential to make a real difference in the development of gene therapies for rare diseases, and I look forward to its application across a broader range of conditions. JCR values its partnership with Alexion, and we are pleased to build on the collaboration we have developed over the years.' The impact of this agreement on JCR's consolidated financial results for the fiscal year ending March 31, 2026, has already been factored into the current earnings forecast. About JUST-AAV JUST-AAV is a proprietary platform technology that utilizes modified adeno-associated virus (AAV) vectors. The technology entails insertion of miniaturized antibodies against receptors on selected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeted delivery to those tissues and organs. Further capsid modifications minimize off-target effects and improve safety. The name is derived from ' J CR' ' U ltimate destination of organ' ' S afeguarding against off-target delivery' and ' T ransformative technology' reflecting its potential for broad application across various diseases. About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO ® (INN: pabinafusp alfa). About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website: Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as 'believe,' 'estimate,' 'anticipate,' 'intend,' 'plan,' 'will,' 'would,' 'target' and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors' pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website
JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website

Yahoo

time06-06-2025

  • Business
  • Yahoo

JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website

- A refreshed global brand signals JCR's expanded international presence and unwavering focus on rare and genetic diseases worldwide - HYOGO, Japan, June 06, 2025--(BUSINESS WIRE)--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 tailored for global audiences that reflects its passion for both science and humanity and brings the JCR brand to life for patients, physicians, and partners worldwide. "At JCR, we are committed to advancing science and delivering on the treatments for patients with unmet needs around the world," said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. "In September, we celebrate the 50th anniversary of the company I founded in 1975. The launch of our new global website reflects our evolution from an innovative startup in Japan to a global biopharmaceutical company. We remain dedicated to developing therapies for people with rare and genetic diseases and partnering with those who share our vision of transforming care for patients and families living with once untreatable diseases." The site features streamlined user interface and curated content for patients, physicians, partners, and investors – reinforcing JCR's commitment to bridge the gap between complex science and unmet needs of patients and families. For the past 50 years, JCR has built a reputation for the development of innovative new therapeutic approaches, including its more recent J-Brain Cargo® technology, a proprietary blood-brain barrier-penetrating technology that can deliver biotherapeutics into the central nervous system (CNS). This platform underpins several investigational pre-clinical and clinical staged therapies in JCR's pipeline, beginning with lysosomal storage disorders (LSDs), and it reflects the company's ongoing commitment to advancing treatments for CNS-related diseases across neurodegeneration, neuro-inflammation, and neuro-oncology conditions. JCR continues to strengthen its global research, development, and manufacturing capabilities, with a focus on addressing unmet medical needs and expanding therapeutic possibilities. Learn more about JCR, its technologies, and clinical development programs on its global website: About the J-Brain Cargo® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO® (INN: pabinafusp alfa). About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website: View source version on Contacts Investors & Media:JCR Pharmaceuticals Co., Communicationsir-info@ Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data

JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website
JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website

Business Wire

time06-06-2025

  • Business
  • Business Wire

JCR Pharmaceuticals Marks 50 Years of Innovation with Launch of One-of-a-Kind Global Website

HYOGO, Japan--(BUSINESS WIRE)-- 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 tailored for global audiences that reflects its passion for both science and humanity and brings the JCR brand to life for patients, physicians, and partners worldwide. 'At JCR, we are committed to advancing science and delivering on the treatments for patients with unmet needs around the world,' said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. 'In September, we celebrate the 50 th anniversary of the company I founded in 1975. The launch of our new global website reflects our evolution from an innovative startup in Japan to a global biopharmaceutical company. We remain dedicated to developing therapies for people with rare and genetic diseases and partnering with those who share our vision of transforming care for patients and families living with once untreatable diseases.' The site features streamlined user interface and curated content for patients, physicians, partners, and investors – reinforcing JCR's commitment to bridge the gap between complex science and unmet needs of patients and families. For the past 50 years, JCR has built a reputation for the development of innovative new therapeutic approaches, including its more recent J-Brain Cargo ® technology, a proprietary blood-brain barrier-penetrating technology that can deliver biotherapeutics into the central nervous system (CNS). This platform underpins several investigational pre-clinical and clinical staged therapies in JCR's pipeline, beginning with lysosomal storage disorders (LSDs), and it reflects the company's ongoing commitment to advancing treatments for CNS-related diseases across neurodegeneration, neuro-inflammation, and neuro-oncology conditions. JCR continues to strengthen its global research, development, and manufacturing capabilities, with a focus on addressing unmet medical needs and expanding therapeutic possibilities. Learn more about JCR, its technologies, and clinical development programs on its global website: About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO ® (INN: pabinafusp alfa). About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world's most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit the global website:

Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment
Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment

Globe and Mail

time20-05-2025

  • Business
  • Globe and Mail

Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment

DelveInsight's, 'Mucopolysaccharidosis Type I Pipeline Insight 2025' report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Mucopolysaccharidosis Type I pipeline landscape. It covers the Mucopolysaccharidosis Type I pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Mucopolysaccharidosis Type I pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Discover the latest drugs and treatment options in the Mucopolysaccharidosis Type I Pipeline. Dive into DelveInsight's comprehensive report today! @ Mucopolysaccharidosis Type I Pipeline Outlook Key Takeaways from the Mucopolysaccharidosis Type I Pipeline Report In May 2025, JCR Pharmaceuticals Co. Ltd announced a JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study. DelveInsight's Mucopolysaccharidosis Type I Pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Mucopolysaccharidosis Type I treatment. The leading Mucopolysaccharidosis Type I Companies such as Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others. Stay ahead with the most recent pipeline outlook for Mucopolysaccharidosis Type I. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Mucopolysaccharidosis Type I Treatment Drugs Mucopolysaccharidosis Type I Emerging Drugs Profile OTL 203: Orchard Therapeutics OTL-203 is an investigational ex vivo autologous gene therapy being investigated for the treatment of MPS-I. It uses a modified virus to insert a functional copy of the IDUA gene into a patient's cells. OTL-203 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-203 has received rare pediatric disease designation from the FDA. Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome. OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority. Currently, the drug is in Phase III stage of its development for the treatment of Mucopolysaccharidosis Type I. JR 171: JCR Pharmaceuticals JR-171, is an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR's proprietary J-Brain Cargo® BBB technology. JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. The trial is also scheduled for enrolling patients in the US. Currently, the drug is in Phase I/II stage of its development for the treatment of Mucopolysaccharidosis Type I. The Mucopolysaccharidosis Type I Pipeline Report Provides Insights into The report provides detailed insights about companies that are developing therapies for the treatment of Mucopolysaccharidosis Type I with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Mucopolysaccharidosis Type I Treatment. Mucopolysaccharidosis Type I Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Mucopolysaccharidosis Type I Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Mucopolysaccharidosis Type I market Explore groundbreaking therapies and clinical trials in the Mucopolysaccharidosis Type I Marketed and Pipeline Drugs. Access DelveInsight's detailed report now! @ New Mucopolysaccharidosis Type I Drugs Mucopolysaccharidosis Type I Companies Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Mucopolysaccharidosis type I (MPS I) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical Mucopolysaccharidosis Type I Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Unveil the future of Mucopolysaccharidosis Type I Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Mucopolysaccharidosis Type I Market Drivers and Barriers Scope of the Mucopolysaccharidosis Type I Market Report Coverage- Global Mucopolysaccharidosis Type I Companies- Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Mucopolysaccharidosis Type I Pipeline Therapies- Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others. Mucopolysaccharidosis Type I Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Mucopolysaccharidosis Type I Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Get the latest on Mucopolysaccharidosis Type I Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Mucopolysaccharidosis Type I Companies, Key Products and Unmet Needs Table of Contents Introduction Executive Summary Mucopolysaccharidosis type I (MPS I): Overview Pipeline Therapeutics Therapeutic Assessment Mucopolysaccharidosis type I (MPS I)– DelveInsight's Analytical Perspective Late Stage Products (Phase III) OTL 203: Orchard Therapeutics Drug profiles in the detailed report….. Mid Stage Products (Phase II) Drug Name: Company Name Drug profiles in the detailed report….. Early Stage Products (Phase I) JR 171: JCR Pharmaceuticals Drug profiles in the detailed report….. Preclinical and Discovery Stage Products Drug name : Company name Drug profiles in the detailed report….. Inactive Products Mucopolysaccharidosis type I (MPS I) Key Companies Mucopolysaccharidosis type I (MPS I) Key Products Mucopolysaccharidosis type I (MPS I)- Unmet Needs Mucopolysaccharidosis type I (MPS I)- Market Drivers and Barriers Mucopolysaccharidosis type I (MPS I)- Future Perspectives and Conclusion Mucopolysaccharidosis type I (MPS I) Analyst Views Mucopolysaccharidosis type I (MPS I) Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 09650213330 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:

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