JCR Pharmaceuticals Highlights Innovative Gene Therapy Research at the 7th International Forum of Lysosomal Disorders
18 July 2024 - 7:28PM
Business Wire
JCR Pharmaceuticals Co., Ltd. (TSE 4552: Chairman and President
Shin Ashida, “JCR”) announced presentations highlighting its latest
advancements in gene therapy research at the 7th International
Forum of Lysosomal Disorders, held in Tokyo on July 12-13,
2024.
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At the forefront of this groundbreaking research is JCR’s
proprietary J-Brain Cargo® technology, modified to the surface of
an adeno-associated virus (AAV) for enhanced central nervous system
drug delivery. Non-clinical trials on mice and monkeys have
showcased the efficient delivery of the AAV vector to the brain.
Unlike traditional AAV vectors that primarily accumulate in the
liver, our new AAV vector utilizing J-Brain Cargo® showed
significantly reduced liver accumulation, effectively targeting the
brain. This breakthrough points towards a potential therapy that
mitigates the risk of liver toxicity commonly associated with AAV
treatments.
"The non-clinical data we have presented demonstrates the
remarkable potential of our proprietary J-Brain Cargo® technology
for gene therapy," said Shin Ashida, Chairman and President of JCR.
"By achieving efficient vector delivery to the brain and reducing
liver-related side effects, we believe our technology can become a
new platform for developing safer and more effective
treatments."
About the International Forum of Lysosomal Disorders
Established in 2007, the forum hosts a platform for global
leaders in genetic disease research to explore and discuss key
areas of lysosomal storage disorders. It aims to foster the
development of internationally acclaimed researchers in Japan.
(https://ifld2024.jp/)
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
developed based on this technology and approved in Japan for the
treatment of MPS II (mucopolysaccharidosis type II) is IZCARGO®
(INN: pabinafusp alfa).
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty
pharmaceuticals company that is redefining expectations and
expanding possibilities for people with rare and genetic diseases
worldwide. We continue to build upon our 49-year legacy in Japan
while expanding our global footprint into the US, Europe, and Latin
America. We improve patients’ lives by applying our scientific
expertise and unique technologies to research, develop, and deliver
next-generation therapies. 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 diseases including MPS I (Hurler,
Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B
(Sanfilippo syndrome type A and B), and more. JCR strives to expand
the possibilities for patients while accelerating medical
advancement at a global level. Our core values – reliability,
confidence, and persistence – benefit all our stakeholders,
including employees, partners, and patients. For more information,
please visit https://www.jcrpharm.co.jp/en/site/en/.
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