Cell and gene therapy
Cell and gene therapy with digital PCR – from research to bioprocessing & QC
The potential of cell and gene therapies to either prevent disease progression or provide a cure offers a great clinical benefit over existing therapies which are often geared toward the treatment of symptoms.
Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. This can include the insertion of a new copy of a gene, gene editing using e.g., CRISPR technology, and gene silencing. Gene therapy is usually performed by viral-mediated gene transfer with Adeno-Associated Virus (AAV) or lentivirus or alternatively by using plasmids. Cell therapy involves the transfer of cells with the relevant function into the patient. The cells are modified outside the body before being injected into the patient, and can come from the patient (autologous cells) or a donor (allogeneic cells). Examples of cell therapy include CAR T-cells.
Over the past decade, cell and gene therapy research has accelerated and there have been several approvals for various disorders, including lymphomas and leukemias, spinal muscular dystrophy and inherited retinal disease. These recent successes are partly due to the rapid advancements in vector biology and drug delivery technology. Nonetheless, these therapies are inherently complex with a large degree of variability. Developing safe and effective cell and gene therapies requires quality control at all stages of the development process. The superior accuracy and precision offered by digital PCR enables you to ensure the quality and reproducibility of your therapeutic product. Not only can you reliably and precisely determine plasmid quality, viral titer and vector copy number, but you can also perform robust contaminant testing, for example, to detect mycoplasma and residual host cell DNA.