Gene Therapy Applications Guide

The rapid growth of gene therapy modalities—including AAVs, mRNA, CRISPR, and oligonucleotides—marks a new frontier in precision medicine, demanding robust analytical control strategies. Reliable, reproducible testing is essential to ensure the quality, safety, and efficacy of these groundbreaking treatments. This guide details cutting-edge chromatographic solutions designed to empower scientists in characterizing these complex modalities with precision, covering everything from capsid integrity to mRNA purity and beyond. You will find specific applications for the analysis of Adeno-Associated Virus (AAV) vectors, including intact capsid protein identification, peptide mapping, and assessing critical quality attributes (CQAs) like aggregation and capsid load. Furthermore, the guide highlights techniques for characterizing mRNA and oligonucleotides, such as new methods for determining mRNA CQAs by enzymatic digestion and LC-MS/MS, aggregate analysis of Cas9 IVT and EGFP IVT mRNA, separation of single-stranded and duplex oligonucleotides, and robust extractions from complex matrices. Learn how selecting the appropriate fit-for-purpose chemistry, such as SEC, Peptide, Intact, and Oligo columns, is crucial to confidently accelerate innovation while maintaining analytical control. Download the full guide for detailed application notes and ordering information.