AstraZeneca’s rare disease unit Alexion has penned a $825 million deal for adeno-associated virus (AAV) capsids from existing partner JCR Pharmaceuticals to develop genomic medicines.
Under the deal, Alexion will be able to use the capsids in up to five of its genomic medicine programs. In return, JCR will receive an undisclosed upfront payment, followed by up to $225 million in potential milestone payments and up to $600 million in sales-based milestones, should all five of the resulting meds make it to market.
JCR will also be in line for tiered royalties on net sales, according to the July 8 post-market release.
The capsids at the center of the deal come from JCR’s AAV vector platform, which is designed to enhance the targeted delivery of gene therapies. Their properties can include avoiding the liver or heading straight to muscle or the brain.
“I am very pleased to announce the signing of this license agreement for our proprietary AAV capsid platform, JUST-AAV,” JCR CEO Shin Ashida said in the release. “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 biopharma markets a range of therapies in its home territory of Japan, including Izcargo, an enzyme replacement treatment for the genetic disorder Hunter syndrome, as well as the growth hormone Growject.
The company has been working with Alexion for a couple of years, with the partnership kicking off in March 2023 based around a potential blood-brain barrier-penetrating neurodegenerative disease therapy. At the end of that year, the partners also agreed to work together on discovering oligonucleotide therapies.
Meanwhile, Alexion scooped up a number of AAV assets in July 2023 as part of a portfolio of preclinical gene therapy programs acquired from Pfizer. At the time, AstraZeneca put that deal in the context of Alexion’s 2022 buy of genomic medicine company LogicBio.