Gene Therapy: From Set-Up To Scale-Up

This eBook aims to bring you the latest developments and leading opinions from key thought leaders in the field.

Gene therapy provides a promising way to treat inherited and acquired diseases via transfer of genetic material. The advancement of viral-vector gene therapies has enabled the industry to evolve and refine manufacturing scale-up approaches to generate viral vectors for a multitude of applications and target cells. With the rapid forward movement of the field as a whole, it is critical to understand and consider variables that can affect scale up and standardization of viral vector production in the commercial setting. Different viral vectors vary in their production timelines, raw material inputs, efficiency and cost-effectiveness. Additionally, viral production platforms can be impacted by gene expression profile differences that exist in different cell lineages, and could benefit from optimization for specific cell line-dependent requirements. It is necessary to ensure that a comprehensive approach to optimizing these variables are factored in when looking at commercial scale-up and implementing viral vector production processes for therapeutic applications.

In this eBook, we delve further into accelerating gene therapy developments, as well as manufacturing solutions, the outcomes, successes, and possible pitfalls in gene therapy research.

This EBook was originally produced by RegMedNet, in partnership with Thermo Fisher Scientific. For more information, visit RegMedNet.com