AstraZeneca’s Alexion Strikes $825M Gene Therapy Deal with JCR Pharmaceuticals for Proprietary JUST-AAV Platform

2025Year7Month8Day, JapanJCRPharmaceutical Company (Tokyo Stock Exchange Code:4552) Announced collaboration with Alexion, AstraZeneca Rare DiseaseAlexionReach a licensing agreement. According to the agreement,AlexionWill ObtainJCRPharmaceuticals proprietaryTheJUST-AAVCapsid platform licensed for the development of genetic medicines. This is the third collaboration between the two parties, once again drawing industry attention to the field of gene therapy.

Core Content of Cooperation

According to the terms of the agreement,AlexionCan beJUST-AAVThe authorized capsid in the platform is used for up to5A gene drug research and development project.

JCRPharmaceuticals will receive an upfront payment and is eligible for a total of up to8.25USD billion in milestone payments. Among these, the highest amount is related to R&D milestones.2.25USD billion, with the highest sales-related milestone amount6Billion USD. The amount is5The total milestone amount after all items have been exercised. In addition,JCRWill also receive tiered royalties based on the product's net sales.

It is worth mentioning that this isJCRAndAlexionThe third collaboration between them. Previously, the two parties had2023Year3MonthlyJ-Brain Cargo®Technology targets neurodegenerative diseases for research collaboration and2023Year12Month collaborates again to explore the discovery of oligonucleotide therapies.

JUST-AAVIs aproprietaryPlatform technology, using a modified adeno-associated virus (AAV) vector. Its technical principle involves inserting small antibodies targeting specific tissues, organs, or receptors on the blood-brain barrier into the capsid surface, thereby enhancing targeted delivery to these tissues and organs. Meanwhile, further capsid modifications can minimize off-target effects and improve safety.

The name is derived from“JCR”“Ultimate destination of organ”“Safeguarding against off-target delivery(Prevent Off-Target Delivery)”And“Transformative technology(Transformative Technology)”`, which also reflects its potential for widespread application across various diseases.`

In addition,JCRPharmaceuticals also developedproprietaryBlood-brain barrier penetration technologyJ-Brain Cargo®, for delivering biologic therapies to the central nervous system. The first drug developed based on this technologyIZCARGO®(Common name:pabinafusp alfa) has been approved in Japan for the treatment of a lysosomal storage disorder.

Screenshot fromJCROfficial Website