Currently, there are three main types of in vitro transcribed (IVT) RNA drugs: conventional, non-replicating mRNA; base-modified, non-replicating mRNA (bmRNA), which incorporate chemically modified nucleotides; and self-replicating RNA (srRNA), based on an engineered viral genome but devoid of viral structural protein genes.
The ability to create copies of the drug after administration enables lower dosing and expands the range of potential indications. However, srRNAs are often much larger in size (9,000-16,000 bases) and traditional biophysical analytical techniques, such as capillary electrophoresis (CE), have been challenging to adapt to these large molecules.
This presentation will showcase how to achieve improved characterization of srRNA using the newest advances in CE. Additionally, we will discuss how these complement and augment in vitro cell-based assays currently being used for potency testing.