Obtain hands-on training in the production of vectors for gene therapy products using an AAV-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.

July 27 – 30, 2027

$4,500 registration fee

Register

Attend and you will learn to 

• 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

Schedule

Day 1	Lectures • Gene Therapy Overview • Viral Vector Structure and Biology • Methods to Produce Gene Therapy Vectors • Single-use Bioreactors Lab Activities • Transient Transfection DOE • Bioreactor Orientation, Rocker Bag Set-up • Inoculation and Infection AAV-GFP Production
Day 2	Lectures • Modes of Bioreactor Operation, Critical Process Parameters and Process Control • Overview of Downstream Processing of Viral Vectors • Vector Harvest Part 1 (Intro and Depth Filtration) • Vector Harvest Part 2 (Centrifugation, Cell Lysis) Lab Activities • Rocker bag monitoring and sampling • 50 L Assembly • Cell Lysis • Depth Filtration for Lysate Clarification
Day 3	Lectures • Ultrafiltration (UF) for Vector Concentration and Buffer Exchange • Intro to AAV Biosafety • Chromatography for Viral Vector Purification Lab Activities • Concentration and Buffer Exchange of Vectors by Ultrafiltration • Affinity Chromatography and Equipment (Column Load)
Day 4	Lectures • Analytical Principles for Gene Therapy • AAV Methods of Quantification Lab Activities • Affinity Chromatography and Equipment (Elution, Data Analysis) • Adherent Cell Culture Scale Up Workshop • Harvest Transient Transfection DOE
	*Subject to change

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 personnel who may not be directly involved with the process they support, including Validation, QA, or QC staff
• Suppliers of components and contractors providing services to companies manufacturing viral vectors

This course was developed with a project award from the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) and financial assistance from the U.S. Department of Commerce, National Institute of Standards and Technology (70NANB17H002).

Additional support has been provided by the North Carolina Biotechnology Center.

Instructors 

• Gary Gilleskie, Ph.D.
• Driss Elhanafi, Ph.D.
• Elissa Hudspeth
• Danny Schmitt
• Suleiman Sweilem