Conversion and condition-resistant regulatory T-cells for immunosuppressive therapy and tissue repair
This technology is a CRISPR gene editing approach to enhance regulatory T-cell (Treg) and CAR Treg stability to create safer and more effective immunosuppressive therapies for transplantation and autoimmunity.
Unmet Need: Improved therapies for immune tolerance in organ recipients
Current immunosuppressive therapies for organ transplantation are not effective in the long term and come with various side effects. Regulatory T cells (Tregs) naturally have immunosuppressive functions, and CAR (chimeric antigen receptor) Tregs can be engineered to tolerate specific antigens, making them a promising approach for treating autoimmune diseases and promoting immune tolerance after transplantation. However, challenges such as CAR Treg instability and low efficacy limit their current use. Improving the stability and function of CAR Tregs could enhance their effectiveness and expand their potential applications.
The Technology: CRISPR-based approach enhancing CAR Treg immunosuppressive ability
This technology uses an RNA-guided nuclease to selectively eliminate or block the expression of specific cell-surface antigens to create regulatory T cells (Tregs) with enhanced stability. These antigens are essential for two key processes: the conversion of Tregs into effector T cells and the binding of antibodies that target T cells, such as anti-CD2. By targeting these antigens, the technology creates Tregs and CAR Tregs that are resistant to both conversion into effector T cells and depletion by T-cell-targeting antibodies. As a result, these Tregs maintain enhanced stability and are more resilient to immunosuppressive treatments. This approach can be used to treat, reduce, or prevent transplant rejection, autoimmune diseases, and graft-versus-host disease by promoting systemic immune tolerance or immunosuppression.
Applications:
- Immune tolerance or immunosuppressive therapy
- Treatment or prevention of autoimmune disease
- Treatment to reduce and/or prevent organ transplant rejection or complications of transplant
- Treat or prevent graft-versus-host disease
- Use of animal-derived organs for transplantation
- Immunotherapy for cancer treatment
- Research model for studying T cell development and function
- Tissue-specific drug delivery
- Treatment for chronic inflammation
Advantages:
- Improves regulatory T-cell (Treg) and CAR Treg function and stability
- Reduces conversion of Tregs into effector T-cells
- Further reduces the risk of organ rejection
- Platform can be modified to engineer other changes to CAR Tregs for application in other diseases
- Enables broader matches between organ donors and recipients
- Minimal immunosuppressive drugs
- Enhanced longevity of Tregs
Lead Inventor:
Patent Information:
Patent Pending (WO/2023/178165)
Related Publications:
Tech Ventures Reference:
IR CU22040
Licensing Contact: Jerry Kokoshka