JCR Pharmaceuticals to Present at the 21st Annual WORLDSymposium™ 2025 in San Diego
JCR Pharmaceuticals Co., Ltd. (TSE 4552), a leading global specialty pharmaceutical company, has announced its participation in the 21st Annual WORLDSymposium™ 2025, which will take place from February 3-7, 2025, in San Diego, California. During this prestigious event, JCR will present valuable findings from its ongoing research and development efforts, focusing on the potential of investigational therapies and its proprietary J-Brain Cargo® technology. This technology enables the delivery of therapeutic agents across the blood-brain barrier (BBB) to treat lysosomal storage and neurodegenerative disorders.
Presentation Overview:
At WORLDSymposium™, JCR will showcase two important presentations:
- Presentation Title: Treatment with a transferrin receptor-targeted β-hexosaminidase A prolongs life span of GM2 gangliosidosis mice
Date/Time: February 4, 2025, 15:30–17:30 PST
Presenter: Kazuki Miyauchi, Scientist, JCR Pharmaceuticals
Poster Number: LB-33 - Presentation Title: Long-term impact of pabinafusp alfa on disease burden in Hunter syndrome: A 4-year follow-up of patient-reported outcomes
Date/Time: February 6, 2025, 15:30–17:30 PST
Presenter: Ana Maria Martins, M.D., Ph.D., Federal University of São Paulo
Poster Number: 222
The first presentation will focus on JR-479, a pioneering investigational enzyme replacement therapy (ERT) that crosses the BBB. JR-479 is aimed at treating GM2 gangliosidoses, a group of severe neurodegenerative conditions. The second presentation will highlight JCR’s lead investigational product, JR-141 (pabinafusp alfa), a fusion protein developed for the treatment of Hunter syndrome, a lysosomal storage disorder.
Highlighting Investigational Therapies:
- JR-479 for GM2 Gangliosidoses:
JR-479 is a recombinant β-Hexosaminidase A enzyme therapy, specifically designed to penetrate the BBB using JCR’s J-Brain Cargo® technology. The therapy aims to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases, both of which result in severe neurodegeneration. These diseases are caused by a deficiency of the enzyme β-Hexosaminidase A, which leads to the buildup of harmful substances in the nervous system. Currently, there are no approved therapies available for GM2 gangliosidoses, making JR-479 a potentially groundbreaking treatment. - JR-141 (Pabinafusp Alfa) for Hunter Syndrome:
The second major focus of JCR’s presentations at WORLDSymposium™ is JR-141, also known as pabinafusp alfa. This recombinant fusion protein combines a humanized anti-transferrin receptor antibody with iduronate-2-sulfatase, an enzyme deficient in people with Hunter syndrome. Pabinafusp alfa utilizes JCR’s J-Brain Cargo® technology to facilitate delivery across the BBB, enabling it to address the central nervous system (CNS) manifestations of Hunter syndrome. This innovative mechanism has shown promise in clinical trials, where it has been linked to improvements in CNS symptoms and a reduction in disease biomarkers.
Approved by the Ministry of Health, Labour and Welfare (MHLW) in Japan in March 2021, pabinafusp alfa is marketed under the brand name IZCARGO® for the treatment of Hunter syndrome, a rare lysosomal storage disorder affecting around 2,000 to 3,000 individuals worldwide. Hunter syndrome leads to a variety of neurological and somatic symptoms, including cognitive decline and progressive physical deterioration. Currently, enzyme replacement therapy is the standard of care, but there are unmet needs related to CNS involvement, which JR-141 aims to address.
The J-Brain Cargo® Technology: A Key Innovation
JCR’s proprietary J-Brain Cargo® technology is central to both JR-479 and JR-141, enabling these therapies to bypass the blood-brain barrier, a challenge that has historically limited the treatment of neurological diseases. This innovative approach allows therapeutic proteins to be delivered directly to the CNS, addressing the core symptoms of diseases like GM2 gangliosidoses and Hunter syndrome.
The first application of J-Brain Cargo® technology led to the approval of IZCARGO® in Japan for the treatment of Hunter syndrome. This breakthrough represents a major step forward in the treatment of lysosomal storage disorders, particularly for CNS-related symptoms that were previously under-addressed.
WORLDSymposium™ 2025: A Platform for Advancing Lysosomal Disease Research
The WORLDSymposium™ is a key global event that brings together researchers, clinicians, patient advocacy groups, and others in the field of lysosomal diseases. It serves as an important platform for the latest advancements in research, drug development, and patient care. JCR’s participation in the symposium will provide an opportunity to share important updates on its ongoing efforts to address rare and genetic diseases.
JCR Pharmaceuticals JCR Pharmaceuticals
In addition to the scientific presentations, JCR will have an on-site booth (#307) at the conference for attendees to learn more about its research pipeline and therapies. This includes insights into the clinical development of JR-479 and JR-141, as well as the innovative J-Brain Cargo® platform.
About Pabinafusp Alfa (IZCARGO®)
Pabinafusp alfa is a promising therapeutic option for patients with Hunter syndrome, particularly those experiencing CNS symptoms. The drug is designed to provide enzyme replacement therapy for iduronate-2-sulfatase deficiency while simultaneously crossing the blood-brain barrier to address neurological manifestations of the disease. Clinical trials have demonstrated its ability to reduce biomarkers associated with CNS involvement and to improve patient outcomes.
About JR-479
JR-479, currently in preclinical development, is being evaluated as a potential treatment for GM2 gangliosidoses. By delivering β-Hexosaminidase A directly to the brain, JR-479 holds the potential to improve patient outcomes in these devastating disorders, which currently lack effective treatments.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. is a global leader in the development of therapies for rare and genetic diseases. The company has a legacy of over 49 years in Japan and is expanding its presence internationally in the United States, Europe, and Latin America. JCR focuses on innovative treatments that address the unmet needs of patients with conditions such as Hunter syndrome, Fabry disease, and other lysosomal storage disorders. Its commitment to improving lives is reflected in its ongoing work to develop next-generation therapies for rare diseases worldwide.
For more information, please visit JCR Pharmaceuticals website.
