Medical Innovation Exchange

Takeda’s gene therapy pivot leaves JCR behind after proof-of-concept preclinical test

JCR Pharmaceuticals has achieved proof of concept for its gene therapy technology—just in time to lose partner Takeda as the pharma pivots away from adeno-associated viruses.

The Japanese companies signed an agreement in March 2022 to work on AAV-mediated gene therapies using JCR’s J-Brain Cargo technology, which aims to penetrate the blood-brain barrier to deliver biotherapeutics to the central nervous system.

Takeda has decided to discontinue the collaboration as it pivots research and development efforts away from AAV-mediated gene therapies, according to a Thursday press release. The company announced the change earlier this year while pledging to continue some investments in non-viral gene therapy. At the time, Takeda said discussions were happening with its gene therapy partners on next steps.

JCR, evidently, now knows the fate of its partnership. The deal existed long enough for the company to receive a milestone payment from Takeda for achieving preclinical proof-of-concept by confirming that the J-Brain Cargo technology had effects on the brain of a rodent model. Now, the patents and results from the preclinical work will return to JCR.

“We are grateful to Takeda for the partnership, and we understand and respect Takeda’s decision to change its R&D focus away from AAV-mediated gene therapies,” JCR Chairman and President Shin Ashida said in a Thursday statement.

JCR will move forward with gene therapy R&D with the hope of finding new partners, Ashida said. The company would like to move beyond lysosomal storage disorders into more neurological diseases.

The termination is expected to have a minor impact on JCR’s consolidated financial results for the fiscal year ending March 31, 2024.

Takeda has poured billions into partnerships with gene therapies over the past few years, totaling about $9 billion in potential biobucks. The pharma walked away from a massive deal with Poseida Therapeutics that involved a hemophilia A therapy in July.

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