Genetically Engineered T Cells with Regnase-1 and/or TGFBRII Disruption for Neurodegenerative Diseases and Beyond

This inventive concept leverages the core technology of genetically engineered T cells with Regnase-1 and/or TGFBRII disruption to target novel applications in neurodegenerative diseases, autoimmune diseases, and cancer stem cell therapy, demonstrating the versatility and broad potential of this innovative approach.

The original patent disclosed genetically engineered T cells with Regnase-1 and/or TGFBRII disruption for improved functionality and persistence in cancer immunotherapy. However, this technology has untapped potential in other fields. Neurodegenerative diseases, autoimmune diseases, and cancer stem cell therapy are areas where targeted therapies are urgently needed. The inventive concept addresses these limitations by adapting the core technology to these new applications.

The inventive concept involves genetically engineering T cells with Regnase-1 and/or TGFBRII disruption to target specific disease mechanisms. For neurodegenerative diseases, the T cells are engineered to express neurotrophic factors, promoting neural cell survival and regeneration. In autoimmune diseases, the T cells are designed to express cytokines that promote immune tolerance, reducing autoimmune responses. In cancer stem cell therapy, the T cells are engineered to express chimeric antigen receptors (CARs) targeted to cancer stem cell antigens and proteins that promote differentiation of cancer stem cells into non-cancerous cells. The CRISPR/Cas-mediated gene editing system is used to disrupt Regnase-1 and/or TGFBRII genes, and the T cells are administered to patients in need or expanded ex vivo using bioreactors and cytokine-supplemented media.

The inventive concept's novelty lies in its application of the core technology to new disease areas, leveraging the improved functionality and persistence of genetically engineered T cells with Regnase-1 and/or TGFBRII disruption. The inventive step involves adapting the gene editing system and T cell engineering strategies to target specific disease mechanisms, demonstrating a non-obvious and innovative approach to addressing unmet medical needs.

Alternative embodiments of the inventive concept include using different gene editing systems, such as base editing or prime editing, to disrupt Regnase-1 and/or TGFBRII genes. Variations may also include using different types of immune cells, such as natural killer cells or dendritic cells, or combining genetically engineered T cells with other therapies, such as checkpoint inhibitors or small molecule drugs.

The inventive concept has significant commercial potential in the fields of neurodegenerative diseases, autoimmune diseases, and cancer stem cell therapy. The market for these applications is substantial, with a growing demand for targeted and effective therapies. The inventive concept's versatility and broad potential make it an attractive opportunity for companies and investors seeking to develop innovative treatments for unmet medical needs.