Enhanced CAR-T Cell Therapy with Regnase-1 and TGFBRII Disruption

A next-generation CAR-T cell therapy system that leverages bioinformatics, CRISPR/Cas-mediated gene editing, and optimized cell culture to generate genetically engineered T cells with improved persistence and functionality, enabling more effective cancer treatment.

The original patent disclosed genetically engineered T cells with Regnase-1 and/or TGFBRII disruption, which demonstrated improved functionality and persistence. However, the original approach relied on manual identification of disruption sites and traditional gene editing methods, limiting its efficiency and scalability. The new inventive concept addresses these limitations by integrating bioinformatics and CRISPR/Cas-mediated gene editing to enable high-throughput, personalized CAR-T cell therapy.

The enhanced CAR-T cell therapy system comprises a bioinformatics platform that identifies optimal Regnase-1 and/or TGFBRII disruption sites for each patient, a CRISPR/Cas-mediated gene editing system that disrupts the identified sites, and a cell culture module that expands and differentiates the genetically engineered T cells. This integrated system enables rapid, high-precision generation of CAR-T cells with improved persistence and functionality, allowing for more effective treatment of cancer.

The new inventive concept introduces the use of bioinformatics and CRISPR/Cas-mediated gene editing to optimize Regnase-1 and/or TGFBRII disruption, which is not disclosed in the original patent. This integration enables a paradigm shift in CAR-T cell therapy, moving from manual, low-throughput approaches to high-throughput, personalized treatments.

Alternative embodiments of the inventive concept could include the use of different gene editing systems, such as base editing or prime editing, or the integration of additional bioinformatics tools to optimize CAR-T cell design. Variations of the system could also include the use of different cell types or the incorporation of additional therapeutic modalities.

The enhanced CAR-T cell therapy system has significant commercial potential in the cancer treatment market, with potential applications in various types of cancer, including leukemia, lymphoma, and solid tumors. The system's ability to enable high-throughput, personalized CAR-T cell therapy could revolutionize the treatment of cancer, providing a significant market opportunity.