JCR Pharmaceuticals Co Ltd (JCR Pharma) manufactures, purchases and sells medical drugs, regenerative medicine products, and pharmaceutical raw materials. The company operates within a single segment: Pharmaceuticals. JCR Pharma’s diverse portfolio includes several significant therapeutic products. Growject is used to treat growth hormone deficiency and short stature. For lysosomal diseases, the company offers Izcargo for Mucopolysaccharidosis type II and Agalsidase Beta BS IV Infusion for Fabry disease. Temcell HS Injection is a regenerative medicine designed to address acute graft-versus-host disease. JCR Pharma provides Epoetin Alfa BS Injection and Darbepoetin Alfa BS Injection to manage renal anemia.

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JCR Pharma is actively engaged in the research and development of innovative pharmaceuticals and regenerative medicine products, focusing primarily on intractable and rare diseases, especially in the pediatric field. The company is leveraging its proprietary blood-brain barrier penetration technology, "J-Brain Cargo," to develop treatments for over 17 types of lysosomal diseases. Key projects include the development of pabinafusp alfa (JR-141) for Hunter syndrome, lepunafusp alfa (JR-171) for mucopolysaccharidosis type 1, and other enzyme preparations for various lysosomal storage disorders. Additionally, JCR is advancing its gene therapy technology, "JUST-AAV," and is involved in collaborative research to develop new gene therapies. The company is also conducting research to expand the applicability of its technologies to various modalities and is focusing on creating new platform technologies. R&D pipeline focuses on advanced therapeutic solutions using its proprietary J-Brain Cargo and J-MIG System technologies. JR-141 is in global Phase III trials for Mucopolysaccharidosis II (Hunter syndrome). JR-171 is in Phase I/II trials for Mucopolysaccharidosis I (Hurler syndrome). Other preclinical projects include JR-441 for Mucopolysaccharidosis IIIA (Sanfilippo syndrome type A), JR-446 for Mucopolysaccharidosis IIIB (Sanfilippo syndrome type B), JR-471 for Fucosidosis, JR-479 for GM2 gangliosidosis, JR-162 is in preclinical development for Pompe disease, and JR-443 for Mucopolysaccharidosis VII (Sly syndrome). In FY2025, the company invested JPY15,431 million in R&D activities, representing 46.7% of its revenue, with an increase of 36.7% YoY.