JCR Pharmaceuticals to Present Data on Lysosomal Storage Disorder Treatments at SSIEM 2024

JCR Pharmaceuticals Co., Ltd. (TSE: 4552), based in HYOGO, Japan, made noteworthy contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024 in Porto, Portugal, from September 3-6, 2024. The company showcased its innovative J-Brain Cargo® technology through two significant presentations focused on therapies for lysosomal storage disorders, highlighting its role in medical advancements.

Shin Ashida, Chairman and President of JCR, emphasized the importance of addressing neurological symptoms in treating lysosomal storage disorders. Ashida noted that their clinical data for JR-171 indicates its potential effectiveness for managing both somatic and neurological symptoms of mucopolysaccharidosis I (MPS I). Additionally, preclinical trials in their GM1 gangliosidosis AAV gene therapy program showed promising results by combining J-Brain Cargo® technology with gene therapy, suggesting a continuous effort to refine these programs.

JCR's presentations included:

JR-171: Mucopolysaccharidosis Type I (MPS I) Poster

This poster highlighted a case report of four individuals with MPS I treated with JR-171. The study, presented by Ana Maria Martins, M.D., Ph.D. from the Federal University of São Paulo, Brazil, detailed neurobehavioral and somatic improvements in patients administered JR-171 intravenously at doses of 2 mg/kg and 4 mg/kg weekly. These patients experienced significant positive changes in their quality of life, illustrating the drug's potential efficacy for MPS I's somatic and neurological symptoms. Further neurocognitive and developmental evaluations are needed to confirm JR-171's effectiveness for neuronopathic symptoms.

GM1 Gangliosidosis Poster

This high-ranked poster, presented by Saki Matsushima, Ph.D. from The Jikei University School of Medicine, Tokyo, Japan, showcased preclinical data from an AAV gene therapy using a mouse model of GM1 gangliosidosis. The study demonstrated the efficacy of AAV expressing anti-transferrin receptor-antibody fused β-galactosidase (TβGal). The AAV, administered intravenously, showed effective CNS penetration and decreased substrate levels in the CNS in a dose-dependent manner. The therapeutic approach, combining AAV and anti-TfR antibody fused biotherapeutics, showed potential for treating hereditary neurological diseases involving the CNS, warranting further evaluation.

Society for the Study of Inborn Errors of Metabolism (SSIEM)

Founded in 1963, SSIEM fosters the study of inherited metabolic disorders. The society's annual international symposium facilitates the exchange of ideas among professionals interested in inborn errors of metabolism.

J-Brain Cargo® Platform Technology

JCR Pharmaceuticals has developed J-Brain Cargo®, a technology designed to enable biotherapeutics to cross the blood-brain barrier and reach the central nervous system. The first drug developed using this technology, IZCARGO® (pabinafusp alfa), has been approved in Japan for treating a lysosomal storage disorder.

Mucopolysaccharidosis I (Hurler, Hurler-Scheie, Scheie Syndrome)

MPS I is a lysosomal storage disorder caused by a deficiency of the enzyme α-L-iduronidase. It results in a wide array of somatic and neurological symptoms and persists as a significant unmet medical need, particularly regarding treatments that address central nervous system symptoms due to the enzyme's inability to cross the blood-brain barrier.

GM1 Gangliosidosis

GM1 gangliosidosis is an autosomal recessive disorder caused by mutations in the GLB1 gene, leading to a deficiency of β-galactosidase and resulting in severe neurodegeneration and systemic disease. The disorder is categorized into three types based on the age of onset.

JR-171

JR-171 is a recombinant fusion protein combining an antibody against the human transferrin receptor with α-L-iduronidase. This design allows the drug to cross the blood-brain barrier, potentially addressing CNS symptoms in MPS I patients. The US FDA has granted JR-171 Fast Track designation.

JCR Pharmaceuticals Co., Ltd.

JCR Pharmaceuticals is a global specialty pharmaceuticals company focused on rare and genetic diseases. Building on nearly five decades of expertise in Japan, the company is expanding its reach globally, including into the US, Europe, and Latin America. JCR is dedicated to developing next-generation therapies to improve patients' lives, with several approved treatments in Japan and investigational products aimed at treating various rare diseases worldwide. The company prioritizes reliability, confidence, and persistence to benefit all stakeholders.

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