JCR Pharmaceuticals Co., Ltd. announced that it has signed a Research Collaboration, Option and License Agreement with Alexion, AstraZeneca Rare Disease for the development of novel oligonucleotide therapeutics to support targeted delivery to certain tissues or organs using J-Brain Cargo, JCR's proprietary blood-brain barrier (BBB)-penetrating technology. Under the terms of the agreement, JCR and Alexion will research and develop oligonucleotide therapeutics using J-Brain Cargo Technology. As part of the collaboration, Alexion may nominate up to three targets in rare disease indications for pre-clinical development using the platform.

Following pre-clinical development, Alexion will have the option to develop and commercialize the potential new drug candidates. The collaboration is the second agreement between JCR and Alexion in 2023, following a March announcement regarding a research collaboration, option and license agreement to develop therapeutic protein candidates using J-Brain Cargo for neurodegenerative disease. JCR will receive an upfront payment from Alexion and has the right to receive certain research, development and commercial milestones and royalties.

J-Brain Cargo technology is JCR's proprietary drug delivery system that efficiently delivers drugs to target tissues, including the central nervous system, through receptor-mediated transcytosis. It is applicable to various modalities including enzymes, oligonucleotides, lipid nanoparticles, gene and cell therapy, peptides, decoy receptors and antibodies.