Latest news with #JCR
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.
Business Wire
07-05-2025
- Business
- Business Wire
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA
TOKYO & HYOGO, Japan--(BUSINESS WIRE)-- 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). MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 1 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the CNS symptoms of this challenging disorder, and is currently being studied in a Phase I/II trial that is being conducted in Japan (JR- 446-101) under a collaboration agreement between the two companies. 'We are extremely pleased that JR-446 has received orphan drug designation from the U.S. FDA,' said Shuichi Watanabe, Representative Director, President and CEO of MEDIPAL. 'We will continue the development of JR-446 in collaboration with JCR so that we can soon deliver this drug to the patients and their families who are desperately waiting, as there is currently no treatment available.' 'This orphan drug designation is an important development step for the JR-446 clinical program,' said Shin Ashida, President, Chairman and CEO of JCR Pharmaceuticals. 'This orphan drug designation supports the potential of this therapy to address the significant unmet medical need of patients with MPS IIIB.' In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement. 2 With the ODD, JR-446 will be eligible for various incentives to encourage the development in the U.S. About Orphan Drug Designation in the U.S. The U.S. FDA's Office of Orphan Products Development grants orphan status to drugs being developed to treat, prevent, or diagnose a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation provides significant incentives to promote the development of the drug including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee. About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families. 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 is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. About MEDIPAL HOLDINGS CORPORATION MEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit About JCR Pharmaceuticals Co., Ltd. JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, JCR is expanding to the US, Europe, and Latin America. JCR's innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. The core values of reliability, confidence, and persistence drive JCR's mission to enhance global medical progress. For more information, 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. References Based on data from JCR's own investigations, referring to the Ministry of Health, Labour and Welfare's public research. Reference: Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
Korea Herald
10-04-2025
- Automotive
- Korea Herald
Top-tier credit ratings fuel Hyundai Card's global push
Leading credit card issuer turns AI into new growth driver Hyundai Card, South Korea's leading credit card issuer, is bolstering its overseas expansion on the back of stronger credit ratings and growing data-driven capabilities. The company recently secured an upgrade from Japan Credit Rating Agency, building on its milestone achievement in November 2023 when it became the first Korean card firm to obtain a JCR rating. In December, JCR raised Hyundai Card's rating one notch to AA- Stable, aligning it with its parent company, Hyundai Motor Group. In February, Hyundai Card also retained its BBB+ Stable rating from Fitch, confirming last year's upgrade from BBB Positive. S&P likewise raised its rating on the firm to BBB+ Stable last year, while Moody's affirmed its rating of Baa1 and stable outlook. The strong external ratings stand out as Korea's economy grapples with persistent inflation, slowing growth and external risks. Hyundai Card's solid financial footing and competitive business model have helped it weather the broader turbulence. Hyundai Card topped Korea's card industry in credit sales last year, with transaction volume climbing 11 percent to 166.27 trillion won ($113 billion), according to the Credit Finance Association of Korea. A key growth engine was its overseas transactions, which surged more than 30 percent to 3.5 trillion won. That pushed its share of overseas spending among local card issuers to 25 percent, further cementing its leadership in the segment. Net income rose 19 percent to 316.4 billion won in 2024, while its user base expanded by 500,000 to a record 12.56 million. The company has also maintained the industry's lowest delinquency rate for the past four years. Fueling Hyundai Card's growth is its distinctive focus on personalized services, powered by data science. Under CEO and Vice Chairman Chung Tae-young digital initiative, the company has invested about 1 trillion won over the past decade to build its own artificial intelligence and data analytics capabilities. That bet resulted in Universe, Hyundai Card's proprietary AI software, which enables hyperpersonalized data analysis and marketing, with applications across industries. Leveraging Universe, the company has come to dominate Korea's private label credit card market, holding a 78 percent share through exclusive partnerships with major brands. More recently, the firm has begun monetizing its technology abroad. In October 2024, it signed a landmark deal to export Universe to Japan's Sumitomo Mitsui Card Co., marking the first software export by a Korean financial company. The company aims to evolve into a broader financial technology player, seeking new global growth opportunities through data-driven collaborations across international markets. Hyundai Card also benefits from the backing of Hyundai Motor Group. Global credit rating agencies have highlighted the card issuer's strategic role within the conglomerate, with Hyundai Card processing about 40 percent of the group's total sales. 'Backed by strong ratings from global credit agencies, we expect to secure stable funding in overseas markets, including through foreign currency bond issuances, while lowering financing costs,' a company official said. 'These favorable ratings will serve as a foundation for our global expansion.'
