Latest news with #JUST-AAV
Business Wire
6 days ago
- Business
- Business Wire
JCR Pharmaceuticals Selected for Japan's Regenerative Medicine CDMO Subsidy Program
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, 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 expand biomanufacturing capacity for regenerative, cell, and gene therapies. Project Overview 1) Project Title JCR Regenerative Medicine CDMO Enhancement 2) Description Building on its expertise in regenerative medicine, JCR will strengthen its biomanufacturing capability through facility upgrades and workforce training. 3) Period Approval date – December 31, 2027 Background and Rationale Since the 2015 approval of TEMCELL ® HS Inj., JCR has steadily built expertise in regenerative medicine while ensuring a stable, long-term product supply. The company is now developing an AAV gene therapy platform, JUST-AAV, based on its proprietary J-Brain Cargo ® technology for delivery across the blood-brain barrier. To leverage these assets as a development and manufacturing partner for regenerative medicine products, JCR applied for this government subsidy to support manufacturing-related capital investment. Looking Ahead With this subsidy, JCR will invest not only in its own pipeline but also in new partnerships, strengthen as its role in biomanufacturing in regenerative and gene/cell therapies. About the Regenerative CDMO Subsidy Program This subsidy program supports the development of domestic CDMO (Contract Development and Manufacturing Organization) facilities and talent to ensure Japan's capacity to manufacture regenerative, cell, and gene therapies. For details, visit: (Japanese only). 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 partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit JCR's 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
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
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.
Business Wire
16-05-2025
- Business
- Business Wire
JCR Pharmaceuticals Presents Preclinical Gene Therapy Data that Demonstrate Promising CNS Uptake at American Society of Gene and Cell Therapy
HYOGO, Japan--(BUSINESS WIRE)-- 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) 28 th Annual Meeting, being held May 13-17, 2025, in New Orleans, LA. In an oral presentation, the JCR researcher reported that the Company's proprietary J-Brain Cargo ® (JBC) technology enables the efficient delivery of an adeno-associated virus (AAV) gene therapy across the blood-brain barrier (BBB) and into the central nervous system (CNS) in mice, monkeys, and several animal models of CNS diseases. We have successfully developed JUST-AAV, a novel AAV vector platform technology designed to enhance targeted delivery and reduce liver tropism, thereby improving safety and efficacy of AAV-based gene delivery technologies to the CNS. JUST-AAV encompasses a range of vector types optimized for various target tissues, including liver-sparing, muscle-targeting, and brain-targeting variants. This proprietary technology holds significant promise for advancing the field of AAV-based gene therapy. 'These findings represent a significant advancement toward new treatments for previously challenging CNS diseases,' said Hiroyuki Sonoda, Ph.D., Director, Senior Managing Executive Officer, and Executive Director, Research Division at JCR Pharmaceuticals. 'Our preclinical data demonstrate that our brain-targeting JUST-AAV technology delivers therapeutic agents to the central nervous system far more efficiently than conventional AAV9, while significantly reducing liver accumulation. This enhanced safety profile greatly increases the potential for clinical translation. This research is a crucial step in our ongoing commitment to developing innovative solutions for unmet medical needs.' JCR showcased the following presentation: Incorporation of transferrin receptor binder and surface mutations into AAV enables efficient brain delivery and reduced liver tropism Presenter: Yuhei Ashida (JCR Pharmaceuticals) Researchers successfully created a brain-targeting AAV vector (brain-targeting JUST-AAV) by incorporating a miniaturized antibody that binds to the transferrin receptor, into the AAV capsid. Furthermore, unique modifications to the AAV capsid sequence significantly reduced AAV vector accumulation in the liver, a known source of adverse effects. In mouse studies, the JUST-AAV vector achieved 77-fold higher expression of green fluorescent protein (GFP) in the brain compared to AAV9, while reducing the tropism to the liver by 99%. To further enhance brain targeting and BBB permeability, the researchers incorporated additional molecules that bind to receptors other than the transferrin receptor into the AAV capsid. In monkey studies, this bispecific vector demonstrated an improved gene delivery efficiency to the brain by several orders of magnitude compared with AAV9, while reducing infection in potentially problematic tissues such as the liver and dorsal root ganglia by more than 90%. Application of JUST-AAV to a mouse model of neuronal ceroid lipofuscinosis resulted in the disappearance of symptoms such as seizures and prolonged lifespan to an extent of a functional cure. These results suggest that the newly developed JUST-AAV technology offers the potential for safer and more efficient gene therapy than conventional gene delivery vectors. About the American Society of Gene and Cell Therapy (ASGCT) The American Society of Gene and Cell Therapy (ASGCT) is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. The mission of the ASGCT is to advance knowledge, awareness, and education, leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. For more information, please visit www. About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo ®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. 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 JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into 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. 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. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit 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.
