A team from BridgeBio, including Matthew Roach, Phillip Wirz, Jeremy Rouse, Allison Schorzman, Clayton Beard, David Scott and other team members, recently published an article describing the work they did using the TESSA® platform to increase their yield of rAAV5.
The article, entitled Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform, can be seen and downloaded in the September 12th issue of Molecular Therapy, Methods & Clinical Development. You can find it online here.
Molecular Therapy Methods & Clinical Development is an international, open-access journal that publishes peer-reviewed research on methods, procedures, and translational advances in molecular therapy. The journal focuses on cell and gene therapy, particularly in how these therapies are applied in clinical settings. Key topics include novel gene delivery methods, the use of integrating and non-integrating vectors, gene editing techniques, and the manufacturing of these products under Good Manufacturing Practice (GMP) conditions. The journal also addresses regulatory science and the commercialization of these innovative therapies.
Article Abstract
Recombinant adeno-associated virus (rAAV) has become a prominent vector for clinical use. Despite an increase in successful clinical outcomes, the amount of high-quality rAAVs required for clinical trials and eventual commercial demand is difficult to produce, especially for genetic diseases that are prevalent or require high doses.
Many groups are focused on establishing production processes that can produce sufficient rAAV while maintaining potency and quality. The authors, a group from BridgeBio Gene Therapy in Raleigh, NC, used a novel production platform to increase their yield of rAAV5. The production platform uses tetracycline-enabled self-silencing adenovirus (TESSA®) to deliver the wild-type AAV replication and capsid genes alongside the adenovirus helper genes necessary for production. In the article, the authors describe their efforts to evaluate the TESSA® platform in house. They conducted numerous experiments to determine the optimal conditions for producing rAAV5 from the TESSA® production system and produced rAAV5 from the TESSA® system to compare against rAAV5 produced from triple transfection.
Ultimately, they generated data that showed that the vector genome yield of rAAV5 produced with TESSA® was >20-fold higher than rAAV5 produced with triple transfection. Additionally, their data show that quality as well as potency in mice of rAAV5 produced with the TESSA® system and by triple transfection are equivalent.
To learn more about TESSA® for scalable AAV production visit our web site or contact us to arrange a time to meet with our technical team.