Cell and Gene Therapy

A diverse range of gene-based therapies and vaccines are being developed today and they are ushering in a new era in genetic medicine.  Different therapeutic approaches include:  

Gene Silencing / Modulation
• Blocking / modulating protein expression (oligonucleotide-based drugs)

Gene Therapy
• In-vivo gene insertion; replacing a faulty gene sequence

Gene Modified Cell Therapy
• Ex-vivo gene insertion; CAR-T, stem cells, allogeneic cells

DNA/RNA Vaccines 
• In-vivo expression of antigens; infectious diseases, cancer immunotherapy

Gene Editing
• Ex-vivo & in-vivo editing; correcting a faulty gene sequence

All require delivery of a nucleic acid sequence (DNA/RNA) into specific cells to work, and many approaches are being developed to enable their safe and effective delivery.  In many ways, the science of achieving targeted drug delivery into specific cells in the body is the new frontier in medicine.

These larger, more complex, multi-component drugs require a range of analytical techniques for characterization and CMC (chemistry, manufacturing and control) monitoring at both the component level (nucleic acids, proteins, lipids, carbohydrates) and at the fully assembled drug product stage to ensure their safety and efficacy. Waters brings industry leading expertise in biomolecular separations, mass analysis, biophysical characterization and compliance-ready informatics, and is a leading provider of the analytical tools and workflow solutions needed to support the development and commercialization of these new and exciting modalities.

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