Sunday, December 10, 2017

We need more viruses!!

Viruses are an essential part of the mechanism of gene therapy delivery systems. Specifically, disabled adenoviruses or lentiviruses are often used to insert the necessary genetic material into the affected cells so that it can exert the desired effects. Unfortunately, these disabled viruses are difficult and costly to produce and gene therapy companies are often unable to do it themselves. Thus, they outsource this step in the production to large biotech firms such as MilliporeSigma, or Oxford BioMedica. There are long waiting lists to obtain viruses from these firms which leads to a back-up in the assembly line of gene therapy production.

Because obtaining these viruses is so expensive, it leads to a major price hike for the getting the actual gene therapy. For example, a gene therapy treatment made by Novartis for a rare blood cancer can cost approximately $475,000 for one dose. Many of these gene-therapy treatments are targeted at extremely rare diseases (oftentimes that only affect about 10 people) so there is much debate surrounding whether or not it is worth the exorbitant cost to produce a treatment that is so limited in scale.

There is a massive amount of therapeutic potential contained within the burgeoning gene therapy research, but we are going to have to find cheaper ways to manufacture them if it is ever going to be a feasible and accessible treatment. The viral vectors are a good place to start.

~Emma

Source: https://www.nytimes.com/2017/11/27/health/gene-therapy-virus-shortage.html

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