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Gene Therapy

Takeda to Explore Gene Therapy Applications of JCR’S J-Brain Cargo Technology

Anna Osborne
29 March 2022

JCR Pharmaceuticals, a global specialty pharmaceutical company based in Japan, has announced an exclusive license and collaboration agreement with biopharmaceutical company Takeda.


The collaboration will see Takeda join the research development of JCR’s proprietary J-Brian Cargo blood-brain barrier (BBB) penetrating technology for treating lysosomal storage disorders (LSDs), providing an opportunity to explore the applications of this technology in other modularity’s, including gene therapy, and to target other conditions of the CNS requiring delivery across the BBB.

JCR developed the novel J-Brain Cargo technology to deliver active drug moleules quickly and efficiently across the BBB to target the CNS. Currently this novel technology has been approved in JCR’s JR-141, for Hunter Syndrome in Japan, and is undergoing Phase III trials in the US and EU. Multiple other clinical and pre-clinical trials of the J-Brain Cargo technology for other LSDs are also underway.

This partnership will see JCR shift focus to develop the next J-Brain Cargo assets to build new game-changing gene-therapy based medicines, utilizing Takeda’s biopharmaceutical global presence and R&D expertise.

Head of Takeda’s Rare Disease Drug Discover Unit, Madhu Natarajan comments that “by combining JCR’s BBB crossing technology with the foundation Takeda is building in gene therapy drug development, we have the opportunity to create a portfolio of gene therapies designed to achieve broad biodistribution including in the central nervous system with the potential to halt or significantly slow progression of the somatic and neuronopathic manifestations of lysosomal storage disorders.”

Under the agreement Takeda will take responsibility for further development and commercialization of any gene therapies to come from the collaboration, paying JCR an upfront sum with supplementary payments for further research, development and commercialization, as well as post-approval sale royalties.

Takeda will also have the ability to nominate additional rare diseases or other disease indications for research. They will then have to pay JCR a fee for each new indication outside the initially focused targets.

“Through this agreement with Takeda, we seek to expand the possibilities of gene therapy using J-Brain Cargo® to develop new gene therapy treatments. I’m pleased to have this opportunity to further our collaboration with Takeda, a highly reputable partner in gene therapy research, and to expand our commitment to making a positive contribution to patients living with lysosomal storage disorders,” comments Shin Ashida, President and Chairman of JCR.

Source: JCR Pharmaceuticals Press Release