Long nucleic acids/transgene sequences in cell and gene therapies range in length from 500 to 10,000 nucleotides or more. Confirming their identity and purity is critically important to ensure the safety and efficacy of these new modalities. Waters chromatography columns and systems combined with optical detection and/or mass spectrometry provide powerful capabilities for confirming the physicochemical properties of these nucleic acids and of their fully assembled drug product; when packaged into lipid nanoparticles (LNPs), viral vectors (e.g. AAV), or virus-like-particles (VLPs) for cell delivery.
From 500 to 10,000+ nucleotides. Leave no detail to chance.
Comprehensive payload profiling: Ensure the safety of mRNA, ssDNA, and dsDNA by assessing integrity, heterogeneity, and purity with integrated LC-MS and light scattering.
Deep mRNA characterization: Verify 5’ capping, 3’ poly-A tailing, and sequence mapping using automated workflows to ensure translation efficiency and stability.
Critical process control: Confirm plasmid linearization efficiency and accurate concentration to prevent premature termination and ensure consistent therapeutic dosing.
Advanced particle analysis: Mitigate safety risks and avoid recalls by identifying subvisible particles using only 5 µL of sample with BMI and FMM technology.
To ensure the stability, efficacy, and safety of cell and gene therapies, it is critically important to assess the integrity, heterogeneity, and purity of their nucleic acid payloads, whether in the form of mRNA, single-stranded DNA (ssDNA), or double-stranded DNA (dsDNA). Chromatography, in combination with light scattering and/or mass spectrometry, is a powerful technique for measuring these critical quality attributes.