Course Description

• This course teaches fundamental concepts in viral vectors for gene therapy and how to manufacture them. The course utilizes case studies and laboratory practices to demonstrate critical steps in the production of viral vectors.

Instructors

• David McNally (MassBiologics)
• Phillip Tai (UMass Medical School)
• Shekar Ganesa (MassBiologics)
• Sangmook Lee (MassBiologics)
• Tom Larkin (MassBiologics)
• Mike Pietila (MassBiologics)
• Gretchen Gee (MassBiologics)
• Matthew Burak (MassBiologics)

Target Audience

• Scientist, Senior Research Scientist, Bioprocess Engineer, or equivalent in Pharmaceuticals, Biopharmaceuticals

Objective

• Understand the major viral vector systems used for gene therapy and their advantages and disadvantages
• Understand the major expression systems used to manufacture viral vectors for use in gene therapy
• Understand the biochemistry of viral vectors, their quality attributes and how to measure them
• Describe some common pitfalls to avoid when processing viral vectors
• Execute transient transfection processes for the production of AAV using suspension HEK293 cells and process the material generated using filtration and chromatographic methods

Modules

• M1. Intro to Viral Vectors and their use in gene therapy
• M2. Upstream processing of Viral Vectors
• M3. Tour of labs and cGMP facility
• M4. Introduction/safety – hands on lab based work
• M5. Hands on lab work – upstream methods
• M6. Downstream processing of Viral Vectors – Chromatography
• M7. Downstream processing of Viral Vectors – Filtration
• M8. Regulatory Considerations for Gene Therapy Products
• M9. Hands on lab work – Downstream Methods
• M10. Analytical Methods
• M11. Analytical Case Studies
• M12. Hands-on lab work – Analytical Methods
• M13. Review of Analytical Data