This course presents an introduction to cell and gene therapy. It covers traditional non-genetic cell therapies, in-vivo gene therapy and ex-vivo gene therapy. Basic genetics and lab techniques are covered. A summary of the AAV manufacturing process for gene delivery is provided. Finally, a summary of the FDA requirements, pathways for approval and long-term concerns for gene therapy is discussed.
Students in biotechnology curriculum program or working within the pharma industry
- Review of Fundamentals
- Key analytical technologies
- Cell therapy (non-genetic, blood and blood products)
- Gene Therapy
- The AAV manufacturing process
- Gene therapy regulations and guidance documents
- FDA pathways for approval
- Concerns
Students who have completed this course should be able to:
- Explain the difference between cell culture, cell therapy and gene therapy. Distinguish between non-genetic and genetic cell therapy (ex-vivo).
- Describe the fundamentals of biotechnology and genetics as they apply to cell and gene therapy.
- Identify the purpose of key analytical equipment used in gene therapy.
- Summarize the gene therapy AAV manufacturing process.
- Describe current regulation/guidance documents on gene therapy.
- Summarize long-term concerns for gene therapy.
Delivered in-person in a classroom or lab setting.
Delivered online with a live instructor at a scheduled date and time.
This course presents an introduction to cell and gene therapy. It covers traditional non-genetic cell therapies, in-vivo gene therapy and ex-vivo gene therapy. Basic genetics and lab techniques are covered. A summary of the AAV manufacturing process for gene delivery is provided. Finally, a summary of the FDA requirements, pathways for approval and long-term concerns for gene therapy is discussed.
Students in biotechnology curriculum program or working within the pharma industry
- Review of Fundamentals
- Key analytical technologies
- Cell therapy (non-genetic, blood and blood products)
- Gene Therapy
- The AAV manufacturing process
- Gene therapy regulations and guidance documents
- FDA pathways for approval
- Concerns
Students who have completed this course should be able to:
- Explain the difference between cell culture, cell therapy and gene therapy. Distinguish between non-genetic and genetic cell therapy (ex-vivo).
- Describe the fundamentals of biotechnology and genetics as they apply to cell and gene therapy.
- Identify the purpose of key analytical equipment used in gene therapy.
- Summarize the gene therapy AAV manufacturing process.
- Describe current regulation/guidance documents on gene therapy.
- Summarize long-term concerns for gene therapy.
Delivered in-person in a classroom or lab setting.
Delivered online with a live instructor at a scheduled date and time.