_Cell Therapy


  • A novel allogeneic CAR-T cell therapy platform using iPSC-derived NKT cells
  • World's first clinical application of iPSC-derived NKT cells in cancer immunotherapy

Induced pluripotent stem cell (iPSC)-derived NKT cells are used as effector for a novel allogeneic CAR-T cell therapy.

NKT cells are immune cells that are capable of directly attacking cancer cells and activating other immune cells via an adjuvant process. Activated NKT cells produce various types of cytokines that elicit activation of natural killer (NK) cells and maturation of dendritic cells of the innate immune system. Mature dendritic cells then cause killer T cells of the adaptive immune system to proliferate and become activated, resulting in a synergistically enhanced anti-tumor effect. By activating innate immune system, NKT cells are capable of killing major histocompatibility complex (MHC)-negative cancer cells, which T cells are incapable of killing. This is one feature that is unique to iPS-NKT cell therapy.

Despite their significant anti-cancer potential, current cell therapies using NKT cells have their downsides. They are extremely difficult to harvest from the body and culture and grow to quantities that are adequate for clinical treatment, since NKT cells make up only from 0.01% to 0.1% of total blood T cells. Overcoming this limitation is what prompted RIKEN to initiate research in iPS cell technology. Dr. Haruhiko Koseki, Group Director of the Laboratory for Developmental Genetics, and associates sought to realize the timely production of NKT cells in sufficient and stable quality and quantity for treatment with iPS cell technology, in which unlimited numbers of NKT cell derived pluripotent stem cells are grown and then induced to re-differentiate into NKT cells.

We have been working on the research and development of this cell therapy together with the developer, RIKEN, and in November 2022, we exercised our in-licensing option right and obtained an exclusive worldwide license to develop, manufacture, and market the product.

With this licensing, we have established a platform consisting of:(i) the basic patent, (ii) the master iPS cell bank (MCB) and (iii) the manufacturing process from iPS cell to accurately differentiated NKT cell. The (i) patent in Japan, the US, and the EU allows BrightPath to stand as the only player licensed to use iPSC-derived NKT cells for allogeneic cell therapy. The (ii) MCB enables BrightPath alone to access the iPSC-derived reproducibility of which clinical safety is being evaluated in the ongoing Phase 1 trial. The (iii) manufacturing process ensures that BrightPath is the sole company having a process capable of differentiating iPS cells into properly functioning NKT cells at high yield and high purity in compliance with the industry regulations.

This platform will serve as a foundation for the development of new gene-modified iPS-NKT cell medicine that introduces CAR genes against cancer antigens in various types of cancer, and will enable the development of such medicine in a wide range of cancer types and in a wide range of regions around the world.

At present, an investigator-initiated Phase 1 trial of iPS-NKT in patients with head and neck cancers is underway at Chiba University as the world's first clinical application of iPSC-derived NKT cells for cellular therapy.

A novel iPSC-derived CAR-invariant natural killer T (iNKT) cell therapy platform for hematologic malignancies and solid tumors

SITC 2022


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