BTEC Short Course: Hands-On cGMP Biomanufacturing of Vectors for Gene Therapy

Why should you attend?

To obtain hands-on training in the production of vectors for gene therapy products using an AAV2-GFP model system in both HEK293 cells and Sf9/Baculovirus systems. Engage in pilot/manufacturing-scale upstream operations (adherent and suspension cell culture, vector production scale-up in cell stacks and single-use bioreactors); downstream operations (cell lysis, product recovery via depth filtration, purification using chromatography, and ultrafiltration for concentration and buffer exchange); and analytical technologies, as well as discuss strategies and issues around scale-up, tech transfer, and adherent vs. suspension cultures.

Attend and you will be able to

• List key cGMP (current Good Manufacturing Practice) requirements/expectations
• Describe the unit operations commonly used for production of gene therapy vectors, including their purpose, underlying principles, and operation
• Culture adherent and suspension cell lines
• Optimize transient transfection conditions
• Harvest vector using scalable operations and describe options for harvest process design
• Purify vector using appropriate resins and AKTA chromatography systems
• Formulate drug substance by ultrafiltration
• Perform basic scale-up calculations for operations involved
• List parameters that are monitored and/or controlled to ensure acceptable process performance
• Calculate step yields for downstream operations
• Describe analytical tools used in assessing and characterizing gene therapy products and processes
   

Who should attend

Anyone interested in obtaining basic knowledge and hands-on exposure to production-scale biomanufacturing operations used in the manufacturing of vectors for gene therapy products, such as the following:

• New or existing biomanufacturing employees taking on a new assignment focused on gene therapy
• Research scientists from academia or industry interested in gaining knowledge of cGMP manufacturing of AAV vectors
• Biomanufacturing support personnel who may not be directly involved with the process they support
• Vendors who supply the biomanufacturing industry with equipment and components