Provides Localized Immune Tolerance to Any Stem Cell-Derived Therapy/Graft or Safety Switch, Allowing for the Elimination of Stem Cell-Derived Grafts
Cell therapy using functional tissues derived from pluripotent stem cells has the potential to revolutionize regenerative medicine by replacing or promoting the repair and regeneration of diseased, dysfunctional, injured or lost tissues. The cell therapy space is experiencing significant growth due to its potential to treat diseases at the genetic level. This presents an opportunity for researchers to address a wide range of conditions. With applications to a wide range of conditions, the global cell therapy market was valued at $5.6 billion in 2024 and is projected to reach $33.93 billion by 2033. However, two main limitations of stem cell-derived cell therapy remain.
First, the need for systemic immunosuppression to avoid allogenic graft destruction. The immunological rejection challenge is further augmented in autoimmune conditions. Secondly, the danger of tumor development; this risk is further heightened when using hypoimmunogenic cells that are genetically engineered to evade the immune system. Currently, it is very difficult to selectively target stem cell-derived grafts. It requires unique cell surface markers to be present and identified that are also uniformly expressed on all stem cell-derived cells within grafts, but not on any other cell within the body of the graft recipient.
Researchers at the University of Florida, in collaboration with the Medical University of South Carolina, have developed a unique surface protein (USP) on stem cells and all cells derived from them. To take advantage of the USP, unique chimeric antigen receptors (CARs) against USP have been developed, a technology that can provide immune tolerance to stem cell-derived grafts via CAR Tregs or effectively destroy stem cell-derived grafts via CAR Teff at will. Importantly, the USP does not exhibit the ability to signal, yet central tolerance exists. These advancements address the shortcomings of current cell therapy efforts by providing an immune tolerance induction approach at the site of engraftment, eliminating the need for systemic immune suppression.
Application
Induces immune tolerance to stem cell-derived grafts, providing unique immune protection in cell therapy
Advantages
- Pluripotent stem cells have a unique target for the induction of immune tolerance or a desired graft, providing immune protection
- Eliminates the need for systemic immune suppression, increasing the patient population that can benefit from curative cell therapy
- Provides an effective safety switch by allowing specific destruction of stem cell-derived grafts, ensuring that patients receive safer, more effective therapies
Technology
This localized immune tolerance induction and safety switch application provides an immune tolerance induction approach at the site of engraftment. This significantly increases the patient population benefiting from curative cell therapy. In contrast to immune evasion approaches, commonly aimed at suppressing only one specific immune axis, immune tolerance addresses multiple axes at once. Notably, this new technology can be readily integrated with existing immune-evasive approaches currently developed in the cell therapy space. Alternatively, this technology provides the prospect of an effective safety switch by allowing specific destruction of stem cell-derived grafts by using USP specific CAR T effector cells, ensuring patients receive safer, more effective therapies. These proprietary modifications in the stem cells, in conjunction with unique CAR constructs, provide unparalleled opportunities for safer, better-tolerated, and functional stem cell-derived cell therapies.
Brochure
