Hyogo, Japan – July 8, 2025 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) todayannounced that it has entered into a license agreement with Alexion, AstraZeneca Rare Disease(“Alexion”), for JCR’s new, proprietary JUST-AAV capsids to develop genomic medicines.
JUST-AAV encompasses a range of vector types optimized for various target tissues—includingliver-sparing, muscle-targeting, and brain-targeting variants—to expand the potential of AAVbased gene therapy.
Under the terms of the agreement, Alexion may use the licensed capsids, which are part of theJUST-AAV platform, in up to five of Alexion's genomic medicines programs. JCR will receive anupfront payment from Alexion. JCR is eligible to receive milestone payments of up to USD 225million related to research and development, and up to USD 600 million related to sales, for atotal of up to USD 825 million (approximately JPY 120 billion converted at the exchange rate ofJPY 145 to USD), which reflects the aggregate milestones if all five targets are exercised. Inaddition, JCR is entitled to receive tiered royalties based on net sales.
This agreement marks the third partnership between JCR and Alexion, following researchcollaborations involving J-Brain Cargo® technology—the first announced in March 2023 forneurodegenerative disease and the second announced in December 2023 for the discovery ofoligonucleotide therapeutics.
“I am very pleased to announce the signing of this license agreement for our proprietary AAVcapsid platform, JUST-AAV” said Shin Ashida, Chairman, President and CEO of JCR. “I believeJUST-AAV has the potential to make a real difference in the development of gene therapies forrare diseases, and I look forward to its application across a broader range of conditions. JCRvalues its partnership with Alexion, and we are pleased to build on the collaboration we havedeveloped over the years.”
The impact of this agreement on JCR’s consolidated financial results for the fiscal year endingMarch 31, 2026, has already been factored into the current earnings forecast.
About JUST-AAV
JUST-AAV is a proprietary platform technology that utilizes modified adeno-associated virus(AAV) vectors. The technology entails insertion of miniaturized antibodies against receptors onselected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeteddelivery to those tissues and organs. Further capsid modifications minimize off-target effectsand improve safety. The name is derived from “JCR” “Ultimate destination of organ”“Safeguarding against off-target delivery” and “Transformative technology” reflecting its potentialfor broad application across various diseases.
About the J-Brain Cargo® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology,J-Brain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developedbased on this technology and approved in Japan for the treatment a lysosomal storage disorderis IZCARGO® (INN: pabinafusp alfa).
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company thatdevelops treatments that go beyond rare diseases to solve the world’s most complex healthcarechallenges. We continue to build upon our 50-year legacy in Japan while expanding our globalfootprint into the U.S., Europe, and Latin America. We improve patients’ lives by applying ourscientific expertise and unique technologies to research, develop, and deliver next-generationtherapies. Our approved products in Japan include therapies for the treatment of growth disorder,MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia.Our investigational products in development worldwide are aimed at treating rare diseasesincluding MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilipposyndrome type A and B), and more. Our core values – Putting people first, Forging our own path,Always advancing, and Committed to excellence – mean that the work we do benefits all ourstakeholders, including employees, partners, and patients. We strive to expand the possibilitiesfor patients while accelerating medical advancement at a global level.