With continued clinical success and increased investment, many gene therapy companies are looking toward manufacturing and commercialization of their lead therapies. The majority of gene therapies in clinical trials utilize viral vectors to carry the therapeutic gene into the target cells. The most common method of making virus involves transfecting mammalian cells with one or more plasmid DNA to generate the viral vector. Once produced and harvested, the vector is then clarified, concentrated, purified, sterile filtered, quality-tested, and stored for use either ex vivo or in vivo depending on the therapy and the virus type being used. There is no “one size fits all” approach for these complex gene therapy products and as such, they can be manufactured using a variety of processes, viral vectors, and cell lines. Among current clinical trials, adeno-associated viruses (AAVs) are used most often.
While gene therapy manufacturing and commercialization strategies have improved significantly over the last five years, there are still several challenges to overcome when looking at how these products can be manufactured consistently, at appropriate scale, and in a cost-effective way. Similar to other biotherapeutics, gene therapy commercialization focuses on managing and balancing risk during decision-making. There are many choices to be made in the clinical, financial, and manufacturing domains and getting all of these to work together cohesively can be a significant challenge. Explore how optimizing your process with an end-to-end integrated solution can reduce costs, increase speed to market and get your final gene therapy product within reach of patients.
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