JCR Pharmaceuticals Co., Ltd., a leading company in the field of specialty biopharmaceuticals that focuses on rare and genetic diseases, has achieved a major research breakthrough through its collaboration with Alexion, a part of AstraZeneca’s Rare Disease division. This development is centered on a novel therapeutic protein candidate aimed at treating a neurodegenerative disorder, utilizing JCR’s unique J-Brain Cargo technology. As a result, Alexion has committed to making a milestone payment to JCR, which has already been factored into JCR’s financial projections for the fiscal year concluding on March 31, 2026.
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The collaboration between JCR and Alexion has not only achieved this significant milestone but also extends to other areas of interest. In December 2023, the two companies agreed to work together on developing oligonucleotide-based treatments using the J-Brain Cargo platform. Furthermore, a license agreement signed in July 2025 supports the development of genomic medicines through JCR’s innovative JUST-AAV gene therapy platform.
The J-Brain Cargo platform is specifically engineered to aid in the delivery of biotherapeutics across the blood-brain barrier, enabling them to reach the central nervous system efficiently. JCR’s first drug developed using this technology, IZCARGO (pabinafusp alfa), has already been approved in Japan for the treatment of a lysosomal storage disorder.
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The JUST-AAV platform, on the other hand, enhances the delivery of gene therapy by incorporating miniaturized antibodies that target specific tissues, organs, or the blood-brain barrier. It also includes modifications to the adeno-associated virus vectors to minimize potential side effects and improve the overall safety profile. The name JUST-AAV captures the essence of the platform, standing for JCR Ultimate destination of organ Safeguarding against off-target delivery and Transformative technology, underscoring its broad applicability across various diseases.
This continuous partnership between JCR Pharmaceuticals and Alexion demonstrates their shared dedication to addressing significant unmet medical needs through advanced and innovative therapies that offer hope for patients suffering from complex neurological and genetic conditions.
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