Achieving therapeutic efficacy with a high performance, electroporation-based cellular engineering technology

Abstract

Background & Aim Advances in cell-based therapies for treating cancer and other diseases have created a demand for rapid and flexible manufacturing processes. While virus-based delivery methods have provided the foundation for the cell therapy revolution, the costs of manufacturing viral vectors and related safety issues have created an urgent need for non-viral approaches to cellular engineering that can be implemented at scale in a GMP environment. In this poster, we present data on a GMP-compliant, scalable electroporation-based technology for engineering primary human cells, stem cells and cell lines with high efficiency while maintaining high cell viability. Methods, Results & Conclusion Examples include introducing precise genomic modifications into primary T cells and hematopoietic stem cells via transfection of mRNA and RNPs encoding zinc finger nucleases and CRISPR/Cas9 components as well as single nucleotide editing for correction of disease causing genetic mutations including X-linked chronic granulomatous disease (X-CGD) and sickle cell disease (SCD). We also include data on redirecting the specificity of T cells and NK cells by expressing recombinant receptors. Finally, we provide examples of cellular engineering with induced pluripotent stem cells (iPSCs) and other types of progenitor cells.