Potency, off-target analysis, and comparability in vivo gene-edited therapy development

The guide RNA is a drug substance or a critical component with its own manufacturing requirement. It is not just a reagent and should not be treated as such. Abi Pinchbeck, Commissioning Editor, Cell & Gene Therapy Insights, speaks with Kok-Seong Lim, Principal, Eight Crest Strategies, about the analytical characterization of gene-edited therapies. They discussed the differences between ex vivo and in vivo approaches, potency assay development, off-target analysis and guide RNA specification, comparability after process changes, release testing, and the emerging technologies such as long-read next-generation sequencing set to reshape how these products are measured.