JCR Pharmaceuticals has signed a licence agreement with Alexion, AstraZeneca Rare Disease, for its proprietary JUST-AAV capsids to advance genomic medicines.
The JUST-AAV platform features a range of vector types optimised for several target tissues, including liver-sparing, muscle-targeting, and brain-targeting variants, enhancing the potential of adeno-associated virus (AAV)-based gene therapy.
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Alexion may utilise the licensed capsids in up to five genomic medicines programmes.
JCR will receive an upfront payment and is eligible for milestone payments of up to $225m for research and development, and up to $600m related to sales. This totals to $825m if all five targets are exercised. Additionally, JCR will receive tiered royalties based on net sales.
This collaboration is the third partnership between JCR and Alexion, following earlier research collaborations involving J-Brain Cargo technology. The first collaboration was announced in March 2023 for neurodegenerative diseases, and the second in December 2023 focused on discovering oligonucleotide therapeutics.
JCR Pharmaceuticals chairman, president and CEO Shin Ashida stated: “I am very pleased to announce the signing of this licence agreement for our proprietary AAV capsid platform, JUST-AAV.
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By GlobalData“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. JCR values its partnership with Alexion, and we are pleased to build on the collaboration we have developed over the years.”
The financial impact of this agreement on JCR’s consolidated results for the fiscal year ending 31 March 2026 has already been included in the current earnings forecast.
JUST-AAV is a proprietary platform technology that employs modified AAV vectors. The technology involves inserting miniaturised antibodies against receptors on selected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeted delivery. Further capsid modifications reduce off-target effects and improve safety.
JCR has also developed a proprietary blood-brain barrier-penetrating technology, J-Brain Cargo, to deliver biotherapeutics into the central nervous system. IZCARGO is the first drug developed with this technology and approved in Japan for treating a lysosomal storage disorder.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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