The deal gives AstraZeneca’s rare disease unit Alexion access to specialized capsids developed by the Japanese biotech JCR Pharmaceuticals for use in up to five of Alexion’s gene therapies.
Alexion, AstraZeneca’s rare disease subsidiary, is licensing specialized gene therapy vehicles from the Japanese firm JCR Pharmaceuticals.
The deal, announced Tuesday, gives Alexion access to JCR’s JUST-AAV capsids to use in up to five of Alexion’s genomic medicines programs. In exchange, Alexion will pay JCR an undisclosed upfront fee plus up to $225 million in milestone payments related to research and development, as well as an additional $600 million in sales milestones. JCR will also get tiered royalties related to sales, though no specifics were given.
JCR shares closed up 15% in the Tokyo Stock Exchange Wednesday.
What indications Alexion would use the capsids for was also not specified. AstraZeneca’s rare disease pipeline is full of mid- to late-stage assets in diseases like myasthenia gravis and lupus nephritis.
The agreement is the third such pact between the two companies, following a March 2023 deal aimed at neurodegenerative diseases and another in December 2023 for the discovery of oligonucleotide therapies. Neither of those deal announcements included financial details, though both made use of JCR’s J-Brain Cargo technology, aimed at ferrying drugs across the blood-brain barrier.
In July 2023, Alexion agreed to pay Pfizer up to $1 billion in potential royalties for a series of assets from Pfizer’s rare disease portfolio, including a suite of novel capsids, though specific target indications were not announced.
JCR boasts that its proprietary JUST-AAV capsids come in several varieties, specifically highlighting “liver-sparing,” muscle-targeting and brain-targeting variants. Injuries to the liver from gene therapies have been under intense scrutiny lately, due to the deaths of two patients from liver injury after receiving Sarepta’s gene therapy for Duchenne muscular dystrophy, Elevidys, earlier this year.
