Latest news with #JCRPharmaceuticals
Globe and Mail
20-05-2025
- 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:
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.
Associated Press
08-05-2025
- Health
- Associated Press
JCR Pharmaceuticals to Present at the American Society of Gene and Cell Therapy 28th Annual Meeting
PRESS RELEASE: Paid Content from Business Wire. The AP news staff was not involved in its creation. Published [hour]:[minute] [AMPM] [timezone], [monthFull] [day], [year] HYOGO, Japan--(BUSINESS WIRE)--May 8, 2025-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; 'JCR') announced today that it will present updated preclinical data from its proprietary J-Brain Cargo ® -applied adeno-associated virus (AAV) vector gene therapy non-clinical research programs in an oral abstract session at the American Society of Gene and Cell Therapy (ASGCT) 28 th Annual Meeting, being held May 13-17, 2025, in New Orleans, LA. The oral presentation details are listed below, and the full program can be found on the ASGCT congress website at . Title: Incorporation of transferrin receptor binder and surface mutations into AAV enables efficient brain delivery and reduced liver tropism Session: AAV Gene Transfer (A): Crossing the Blood-Brain Barrier Abstract Number: 92 Date/Time: Wednesday, May 14, 2:30-2:45pm CT Presenter: Yuhei Ashida, Associate Senior Scientist, at JCR Pharmaceuticals 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. 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. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. For more information, please visit . View source version on CONTACT: Investors & Media: JCR Pharmaceuticals Co., Ltd. Corporate Communications [email protected] KEYWORD: UNITED STATES JAPAN NORTH AMERICA ASIA PACIFIC LOUISIANA INDUSTRY KEYWORD: SCIENCE NEUROLOGY OTHER SCIENCE RESEARCH PHARMACEUTICAL GENERAL HEALTH HEALTH GENETICS SOURCE: JCR Pharmaceuticals Co., Ltd. Copyright Business Wire 2025. PUB: 05/08/2025 12:05 PM/DISC: 05/08/2025 12:06 PM
