JCR Pharmaceuticals unveiled significant advancements in gene therapy research at the International Forum of Lysosomal Disorders. The company’s proprietary J-Brain Cargo® technology, modified to enhance drug delivery to the central nervous system, showed promising results in non-clinical trials.
The modified adeno-associated virus (AAV) vector successfully delivered therapeutic genes to the brains of mice and monkeys. Unlike traditional AAV vectors, which accumulate in the liver, J-Brain Cargo® significantly reduced liver toxicity.
Established in 2007, the forum brings together global leaders in lysosomal storage disorder research. Its mission is to foster international collaboration and advance research in this field.
JCR Pharmaceuticals’ J-Brain Cargo® technology enables the penetration of biotherapeutics into the central nervous system, crossing the blood-brain barrier. IZCARGO®, approved in Japan for treating MPS II, is the first drug developed using this technology.
JCR Pharmaceuticals, a global specialty pharmaceutical company, focuses on rare and genetic diseases. With its headquarters in Japan, the company operates in the US, Europe, and Latin America. JCR’s approved products include treatments for growth disorders, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. Its pipeline includes MPS I, MPS II, and MPS IIIA and B investigational products.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
