Democratizing Gene-Modified Cell Therapy Through Lipid-Based Transfection

Gene-modified cell therapies (GMCTs), such as engineered T cells, Natural Killer (NK) cells, and Hematopoietic Stem Cells (HSC), are at the forefront of treatments for cancer, autoimmune diseases, and genetic disorders. Traditionally, viral vectors have been the backbone of gene transfer and cell editing, but they are often associated with prolonged manufacturing timelines, elevated costs, and safety concerns. Electroporation, another common method, is also linked to elevated costs as well as lower cell viability, which impedes productivity. More recently, lipid nanoparticles (LNPs) have come to the forefront as a method to efficiently transfect immune and stem cells ex vivo. While this newer method has addressed many challenges, it still presents with high upfront costs to manufacturing, and lengthy development processes. To address these challenges, we have developed a pre-formed lipid nanoparticle as a promising alternative for ex vivo transfection of immune and stem cells. This technology is applicable with various forms of RNA, and DNA, and allows for rapid production of complexes. Here, we will introduce this innovative, non-viral delivery solution, showcasing its efficiency in producing CAR-T and CAR-NK cell therapies, while minimizing manufacturing costs, development time, and harm to cells.