Progress to commercialization of gene therapy

Human Gene Therapy Journal – The success of gene therapy will depend on the ability to advance viral delivery vectors to commercialization

At an ever-increasing pace, there is promising news regarding clinical results using investigational gene therapy products is emerging, including several exciting advances with recombinant adeno-associated virus (AAV).

Yuan and coworkers present an improved and simplified method for generating producer cell lines that yield large amounts of AAV and exhibit stable growth, in “A Versatile Adeno-Associated Virus Vector Producer Cell Line Method for Scalable Vector Production of Different Serotypes.”

They report improvements and simplifications to previously reported HEK293 based producer cell lines. In this system AAV vector generation can be supported using a replication-deficient E1-deleted adenovirus, because HEK293 cells already contain the adenoviral E1 gene, resulting in highly efficient production of AAV vectors (more than 50 trillion vector genomes per CF10 Cell Factory). This new technology further broadens options for large-scale vector production in a safe and scalable manner.

Other researcher are proving out strategies for minimizing the unwanted production of AAV capsid protein by contaminating replication-competent virus that can provoke an immune response.

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