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Manufacturing viral gene therapy vectors: general approaches and challenges

Published on August 5, 2014   36 min

Other Talks in the Series: Gene Transfer and Gene Therapy

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0:00
Hello. And welcome to the next installment of the Henry Stewart Recorded Lecture Series on the science of gene therapy. My name is John Gray. Today I'll be providing a general overview of the science of manufacturing viral gene therapy vectors.
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The manufacturer of viral gene therapy vectors is essentially a co-opting of the natural virus life cycle to selectively harness the natural power of viruses to deliver genes to cells while avoiding the pathological consequences of a natural viral infection. Virologists typically study viruses in the context of a life cycle that begins when a viral particle infects a naive cell. This converts the naive cell into a virally infected cell, and as natural viruses contain all the genetic information necessary to instruct the cell to replicate the virus, this effectively creates a virus-producing cell. Because this cycle begins with the infection event, virologists typically describe this infection process as the early phase, and the process whereby viral particles are produced from a virally infected cell as the late phase.
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Manufacturing of viral gene therapy vectors is therefore an attempt to mimic the late phase of the viral life cycle in a clean room environment in order to make a product which can be used later to perform the early phase by delivering genetic cargo to a patient's cells. For an injectable vector, viral vector particles are generated from vector producer cells in a clean room and are the final vialed drug product.
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Manufacturing viral gene therapy vectors: general approaches and challenges

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