After decades of hope and hype, the first engineered cell and gene therapy (CGT) products were approved by the FDA in 2017. Several more approvals followed in quick succession marking the beginning of a new wave in treatment modalities for patients. CGT products can transform how certain diseases are treated and can offer life-extending and potentially curative benefits for patients. Cells such as stem cells and immune cells can be ‘engineered’ or modified by delivering DNA, RNA, proteins or gene editing tools such as Crispr Cas9 into the cells to add or remove functionality. The engineered cells are then administered to patients for specific therapeutic purposes. The intracellular delivery step has traditionally been carried out using viral vectors, but this has many challenges and limitations. This has driven interest in non-viral methods for intracellular delivery. This presentation will outline developments in the CGT field and will describe a non-viral delivery technology for cell engineering that was developed in Avectas, a Maynooth University spin-out company.
