Cell and gene therapies have the potential to provide long-term therapeutic efficacy, but their development is both risky and cost-intensive. Obtaining a better understanding of the specific structural attributes of vector proteins that influence clinical delivery and outcomes is essential for advancing both individual candidates and the platforms on which they are based.
Strategic application of advanced analytical techniques can help improve the selection of candidates and guide development decisions, leading to a greater number of safer, more efficacious cell and gene therapies successfully completing development and entering the market. High-resolution LC-MS based characterization, quantification, and in vivo expression profiling approaches are increasingly being applied to enable improved program understanding, controls, and clinical outcomes assessment.