Potency, off-target analysis, and comparability in 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.

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