Let’s Focus on Filtration
No patient has contracted a viral infection from a recombinant product – but, with fewer options downstream, gene therapy manufacturers will need to focus on upstream filtration to achieve the same success
Morven McAlister, Aernout Martens | | Opinion
Gene therapies are an exciting new class of treatment that alters the genetic composition of cells to correct disease-causing mutations – offering patients new therapies for previously untreatable genetic diseases. In 2017, the FDA approved its first gene therapy; at the time of writing, there are 17 approved cell and gene therapies, with many more on the way (1).
Although the pace of discovery is promising, it is only half the battle. Without robust manufacturing processes, these therapies will remain out of reach for the vast majority of patients. Viral vectors, used to transport therapeutic genetic material into cells, must be effectively processed and purified for clinical use. Manufacturing viral vectors for rare diseases already poses challenges, even if the number of patients requiring treatment – and therefore the volume of viral vectors – is low. Therapies for more common diseases such as Alzheimer’s, which impacts at least 50 million people worldwide, would be even more challenging to manufacture (2).
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