Latest news with #Alexion
Yahoo
08-07-2025
- Business
- Yahoo
JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines
HYOGO, Japan, July 08, 2025--(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-AAVJUST-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 "JCR" "Ultimate destination of organ" "Safeguarding against off-target delivery" and "Transformative technology" reflecting its potential for broad application across various diseases. About the J-Brain Cargo® Platform TechnologyJCR 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., 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 StatementsThis 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. View source version on Contacts Investors & Media:JCR Pharmaceuticals Co., Communicationsir-info@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Associated Press
08-07-2025
- 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
Business Wire
08-07-2025
- 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.
Yahoo
25-06-2025
- Business
- Yahoo
Essential Pharma strengthens Board with the appointment of Nicola Heffron as Non-Executive Director
Essential Pharma strengthens Board with the appointment of Nicola Heffron as Non-Executive Director Egham, UK – 25 June 2025 – Essential Pharma ('Essential' or 'the Company'), a global pharmaceutical company developing and delivering medicines for patients in niche populations, today announces the appointment of Nicola Heffron as a Non-Executive Director with immediate effect. Nicola has over 25 years of experience across the pharmaceutical and biotech industries, with a focus on rare diseases and oncology. She is currently Senior Vice President, Global Marketing & Market Access at Alexion, where she leads global commercial strategy, marketing, pricing and market access. Nicola previously held various senior roles at bluebird bio and related businesses, including Chief Operating Officer of bluebird bio Oncology, as well as Chief Operating Officer at the bluebird bio spin-out 2seventybio. Her earlier experience includes senior positions at Celgene, Shire, GSK, and Eli Lilly, specialising in oncology and respiratory diseases. Nicola holds a Bachelor of Pharmacy degree from Bradford University and an MBA from Warwick University. Lee Morley, Chairman of the Board at Essential Pharma, said: 'Nicola joins the Board at an important moment in Essential Pharma's journey. In particular, we look forward to benefitting from her insights as we seek to expand our rare disease footprint, as well as growing our portfolio and development pipeline more broadly.' Emma Johnson, CEO of Essential Pharma, added: 'I'm proud to welcome someone of Nicola's calibre to Essential Pharma's Board. Her extensive leadership experience across global pharmaceutical and biotech companies, and her focus in rare diseases and oncology, will be invaluable as we work to make a difference for patients in niche patient populations.' About Essential Pharma Essential Pharma is a global pharmaceutical company developing and delivering medicines for patients in niche populations. We have a proven track record of acquiring, investing in and commercialising treatments. Our growing product portfolio reaches patients in approximately 70 countries, and we cover multiple therapy areas with a particular focus on rare disease, CNS, ophthalmology and gastroenterology. Our first development-stage asset is an anti-GD2 antibody for the treatment of high-risk neuroblastoma. We challenge convention and work smarter to help ensure patients in small, underserved or rare disease populations have access to the medicines they need. Every patient matters. For more information, visit CONTACTS Essential Pharma Emma Johnson, CEO Tel: +44(0)1784 477 167 Email: info@ ICR Healthcare Tracy Cheung/Chris Welsh/Lucy Featherstone Tel: +44 (0) 20 3709 5700 Email: Essentialpharma@
Newsweek
16-05-2025
- Business
- Newsweek
The New Era of Life Sciences: The Future is Now
When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal dystrophy—had only hope that one day they might see the world. Today, thanks to an investigational gene therapy, we know that the 11 children who participated in the trials can, for the first time in their lives, discern a toy, recognize a human face and even spot a grain of sand. The biotech behind this achievement is MeiraGTx, which has now filed its therapy for approval under exceptional circumstances in the U.K., which would expedite the process. Stories like that of MeiraGTx illustrate the transformative power of gene therapies on patients. More broadly, they highlight the potential for rare diseases R&D to emerge as the principal driver of therapeutic innovation. "Rare diseases have become a critical area for exploring new treatment modalities because, in many cases, there is no existing therapy, or even a model. For patients who have no options, innovative approaches are important," shares Marc Dunoyer, the CEO of Alexion, the rare diseases subsidiary of AstraZeneca. Ironically, the very severity and uncommonness of these illnesses can also be a blessing, as it allows for bolder approaches by both researchers and regulators. Moreover, rare disease drugs have historically had higher approval success rates compared to other drugs. The Tufts Center for the Study of Drug Development found that orphan-designated drugs had a Phase-1-to-approval success rate of 17 percent, compared to 7.9 percent for non-orphan drugs. Benefiting from faster approval pathways and enhanced regulatory incentives, entrepreneurs in the field face better odds of innovating successfully. And when they do, the implications of their therapies go far beyond their original target. "Many of the advances seen today, such as gene editing and RNA-based therapies, originated in rare disease research before moving into larger indications," says Dunoyer. Arcturus Therapeutics, for instance, is addressing cystic fibrosis via a new delivery mechanism. "Our cystic fibrosis program is a prime example, where the ability to inhale mRNA safely could have a huge impact on the pharmaceutical industry. The rare disease field offers a shorter regulatory pathway to approval and a higher likelihood of success. This makes it an attractive space for evaluating next-generation technologies like mRNA therapeutics," shares Arcturus' CEO, Joseph Payne, as the company is expecting results from their Phase 2 trials in 2025. Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics. Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics. We can perform comprehensive genomic profiling to uncover the molecular basis of a tumor. This allows doctors to provide tailored treatments. Over time, this will enable us to move to a future where we understand the molecular drivers for cancer and can deliver truly personalized healthcare. Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina. Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina. Whether it is providing clarity for families dealing with rare genetic disorders or enabling rapid and precise cancer diagnoses, our goal is to ensure patients receive the answers they need when they need them. This report has been paid for by a third party. The views and opinions expressed are not those of Newsweek and are not an endorsement of the products, services or persons mentioned. Click here to download the full report
