Gene therapy involves inserting a new section of DNA into a patient’s cells, often encoding a healthy gene to replace a disease-causing copy. This DNA is delivered by viral vectors which are usually built from plasmids. Plasmids are small sections of genetic material which can be combined to contain all the genes necessary to deliver the gene therapy.
Adeno-associated virus (AAV) and lentiviral plasmids are commonly used for gene therapy delivery. Many challenges associated with manufacturing therapies on a large scale are caused by difficulties in working with plasmids. When manufacturing a plasmid, the final product must be consistent across batches and must not have altered genetic material or other variations that affect quality. Often, small-scale manufacturing processes using adherent cells require significant expertise and process optimisation to adapt to suspension culture for clinical or commercial use. There are also regulatory and timescale challenges associated with plasmid production which delay drug development.
In this article, we will examine how processes for the design and manufacturing of AAV and lentiviral plasmids can be constructed to meet the quality, regulatory and timescale challenges of bringing a new drug to market.
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