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rAAV gene therapy: manufacturing, analysis & comparability

Published on June 30, 2026   27 min

A selection of talks on Pharmaceutical Sciences

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0:00
Hello. My name is Dr. Courtney Sloan, and I'm a Senior Principal Scientist at Pfizer, actually located in the Midwest, and I've spent the last seven years working on our gene therapy products. So I'll begin our topic, Adeno-associated virus gene therapy challenges in manufacturing and analytical processes. I will then hand over the talk to Dr. Cheng for a discussion on establishing comparability when these processes change.
0:30
As the outline here for this talk suggests, I'll begin our topic with a manufacturing process overview highlighting the main areas for development, optimization and challenges from upstream proceeding through downstream purification. Following that, we'll transition to a discussion on the challenges in developing for main analytical assays, for critical quality attributes unique to gene therapies and a discussion on establishing comparability when these processes change.
1:03
Adeno-associated virus manufacturing at the highest level has three main requirements, the production of AAV Cap and Rep genes, the production of the therapeutic gene of interest, which is flanked by ITRs on either end, as well as helper genes. Various challenges exist throughout this manufacturing process, from the choice of cell line, manufacturing platform, through the subsequent purification steps. Many of these process components are capsid serotype, and even at times, trans gene dependent.
1:37
Manufacturing options within the AAV space are still rather diverse, with both transient and stable or hybrid cell banks available for use. The most commonly implemented at this time is the HEK293 cells transfected with plasmids, and for the subsequent discussion, this will be the focus. By far, the largest challenge to

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rAAV gene therapy: manufacturing, analysis & comparability

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