HYOGO, Japan – July 7, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552) today announced it has entered into a license agreement with Alexion, AstraZeneca Rare Disease, for JCR’s novel, proprietary JUST-AAV capsid platform to advance the development of genomic medicines. This agreement marks the third partnership between JCR and Alexion, building on previous collaborations for J-Brain Cargo technology.
The JUST-AAV platform encompasses a range of modified adeno-associated virus (AAV) vectors that are optimized for highly targeted delivery to various tissues. These include liver-sparing, muscle-targeting, and brain-targeting variants, designed to significantly expand the potential and improve the safety profile of AAV-based gene therapies. The technology utilizes the insertion of miniaturized antibodies against specific receptors on selected tissues, organs, or even the blood-brain barrier onto the capsid surface, thereby enhancing targeted delivery and minimizing off-target effects.
Under the terms of the agreement, Alexion gains rights to use the licensed capsids in up to five of its genomic medicines programs. JCR will receive an upfront payment and is eligible for substantial milestone payments totaling up to USD 825 million (approximately JPY 120 billion) if all five targets are exercised. This includes up to USD 225 million related to research and development milestones and up to USD 600 million tied to sales milestones. Additionally, JCR will receive tiered royalties based on net sales of any approved products.
Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals, expressed his enthusiasm, stating, “I believe JUST-AAV has the potential to make a real difference in the development of gene therapies for rare diseases, and I look forward to its application across a broader range of conditions.”
The financial impact of this agreement for the fiscal year ending March 31, 2026, has already been incorporated into JCR’s current earnings forecast.
Source:
https://www.biospace.com/press-releases/jcr-pharmaceuticals-enters-license-agreement-with-alexion-for-proprietary-just-aav-capsids-to-be-used-in-the-development-of-genomic-medicines
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related Services
-
Viral Vector GMP Manufacturing
Empowering Gene Therapy from Concept to Commercialization
-
AAV Packaging – Research Grade
Comprehensive AAV Solutions, From Discovery to Delivery
-
AAV Analytical Services
Comprehensive AAV Solutions, From Discovery to Delivery
-
AAV Capsid Engineering
Proven technology paving your path to effective therapies for cancer or genetic disorder
-
AAV Library Service
At PackGene, we offer a comprehensive range of AAV library services designed to advance your gene therapy research. Our expertise in AAV library creation and screening allows us to provide solutions tailored to your specific needs, enhancing gene delivery specificity and efficiency while minimizing off-target effects and bypassing innate immune responses.