Japan’s JCR Pharmaceuticals will license its new, proprietary JUST-AAV capsids to Alexion, AstraZeneca Rare Disease in a potential $825 million deal.
Per the agreement, Alexion may use the licensed capsids, which are part of the JUST-AAV platform, in up to five of its genomic medicines programs. JCR will receive an upfront payment from Alexion, as well as milestone payments of up to $225 million related to R&D, and up to $600 million related to sales — for a total of up to $825 million.
JCR's JUST-AAV encompasses a range of vector types optimized for various target tissues — including liver-sparing, muscle-targeting, and brain-targeting variants — to expand the potential of AAV-based gene therapy. The technology entails insertion of miniaturized antibodies against receptors on selected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeted delivery to those tissues and organs.
The agreement marks the third partnership between JCR and Alexion. The pair signed research collaborations involving JCR’s J-Brain Cargo technology in March 2023 for neurodegenerative disease and in December 2023 for the discovery of oligonucleotide therapeutics.
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