Rare Daily Staff
JCR Pharmaceuticals announced that it has entered into a license agreement with Alexion, AstraZeneca Rare Disease, for JCR’s proprietary JUST-AAV capsids to develop genomic medicines.
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 uses miniaturized antibodies against receptors on selected tissues, organs, or the blood-brain barrier onto the capsid surface, to enhance targeted delivery to those tissues and organs. Additional modifications to the capsid minimize off-target effects and improve safety.
Under the terms of 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 for an undisclosed amount. JCR is eligible to receive milestone payments of up to $225 million related to research and development, and up to $600 million related to sales, for a total of up to $825 million if all five targets are exercised. In addition, JCR is entitled to receive tiered royalties based on net sales.
The agreement marks the third partnership between JCR and Alexion, following previous research collaborations involving J-Brain Cargo technology—the first announced in March 2023 for neurodegenerative disease and the second in December 2023 for the discovery of oligonucleotide therapeutics.
“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,” said Shin Ashida, chairman, president, and CEO of JCR Pharmaceuticals.
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