Cell and Gene Therapy

Within these therapies lies a diverse range of therapeutic approaches all aimed at treating or curing disease by either blocking, altering or augmenting the expression of specific genes.

What is Gene Therapy​?
Gene therapy is the in vivo delivery of a nucleic acid sequence where multiple modalities exist, but in general, they can be grouped into three categories: 1) gene silencing/modulation 2) gene insertion/replacement and 3) gene editing. Delivery is typically addressed via encapsulation in viral vectors (ie. adeno-associated virus (AAV)), or lipid nanoparticles (LNPs), which become an integral part of the drug product and must be fully characterized as well.

What is Cell Therapy?
Cell therapy is when the nucleic acid is delivered into cells ex vivo and these engineered cells are subsequently delivered into a patient. Both autologous and allogeneic cell therapies are being developed today.

At their core, cell and gene therapies are comprised of nucleic acids, proteins and lipids that need to be fully characterized and for which precise chemistry, manufacturing and control (CMC) processes must be established to ensure their safety and efficacy. Waters offers a broad set of tools, solutions and expertise in biomolecular separations, mass analysis and biophysical characterization to support the development and commercialization of these new and exciting modalities.

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