JCR Pharmaceuticals Co., Ltd. has entered into a Research Collaboration, Option, and License Agreement with Alexion, AstraZeneca Rare Disease, to develop new oligonucleotide therapeutics for rare diseases. The collaboration will leverage JCR’s proprietary J-Brain Cargo® technology, which facilitates the penetration of the blood-brain barrier (BBB).
Under the terms of the agreement, JCR and Alexion will work together to create oligonucleotide therapeutics for up to three rare disease targets using JCR’s J-Brain Cargo® platform. Alexion can take promising pre-clinical drug candidates forward into development and commercialization.
Shin Ashida, Chairman and President of JCR, expressed enthusiasm for the expanded collaboration with Alexion, reinforcing JCR as a partner of choice in developing targeted delivery biotherapeutics. The versatile J-Brain Cargo® technology can deliver drugs efficiently to target tissues, including the central nervous system (CNS), and is compatible with various modalities such as enzymes, oligonucleotides, and gene therapy, among others.
This collaboration follows a 2023 agreement between JCR and Alexion focusing on developing therapeutic protein candidates using J-Brain Cargo® for neurodegenerative diseases. JCR will receive an upfront payment and is eligible for additional research, development, commercial milestones, and royalties.
IZGARGO® is the first BBB-penetrating drug developed with J-Brain Cargo® technology and approved in Japan for a lysosomal storage disorder. JCR will initiate clinical trials for four additional programs using this platform by FY2028.
JCR is a specialty pharmaceutical company with a history of over four decades, committed to delivering innovative treatments for individuals with rare and genetic diseases, continually pushing the boundaries of what’s possible in medical science and therapy.
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