Gene Therapy Manufacturing Primer 101

Gene Therapy Manufacturing Primer 101 is a foundational class that ventures into the essential AAV production platforms, comparing four different approaches and dissecting the key elements of the AAV cassette. This class illustrates the basic upstream/downstream bioprocessing stages, contrasting suspension and adherent cell lines. Bioreactors, such as hyperstacks and iCellis, are examined for their role in this cutting-edge process. This primer ends by summarizing the role of CMC in AAV development, giving an overview of identity, potency, purity, and sterility testing required by the regulatory authorities. Grab your seat today for this introductory course and become conversant in gene therapy manufacturing! 

Section 1: AAV Properties

Explore the structures, functions, and tissue tropism of popular AAV serotypes, gaining a deep understanding of AAV characteristics.

Section 2: AAV Production Platforms

Compare four AAV production platforms, delve into key features of AAV vector DNA, understand the AAV cassette, and explore the production of cell banks, including master and working cell banks, as well as AAV double-stranded and single-stranded DNA.

Section 3: AAV Upstream Bioprocessing

Explore the stages of AAV manufacturing, including the use of suspension and adherent cell lines, learn about upstream bioprocessing steps, small and large batch production, and discover various bioreactors such as hyperstacks and iCellis.

Section 4: AAV Downstream Bioprocessing

Learn more about cell harvesting, downstream bioprocessing, and purification platforms, including chromatography, filters, and centrifugation. Explore fill and finish processes, packaging, and gain an overview of the AAV viral vector manufacturing workflow.

Section 5: AAV CMC

Explore the critical role of Chemistry, Manufacturing, and Controls (CMC) in AAV manufacturing, covering ICH guidelines, identity, potency, quality, purity, and sterility testing. Gain insights into CMC regulatory considerations and the unique challenges in AAV manufacturing.

Five Takeaways


  1. Gain mastery in the structure and function of AAV.
  2. Describe how AAV types affect tissue targeting and give examples.
  3. Compare and contrast common AAV vector systems./li>
  4. List in order the general steps of AAV gene therapy production.
  5. Investigate the main regulatory guidelines for conducting CMC evaluations of AAV viral vectors.

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