Gene Therapy Manufacturing Primer 201

Gene Therapy Manufacturing Primer 201 is a deep dive into cGMP manufacturing processes for AAVs. This class explores in detail the science and regulatory controls of AAV gene therapy manufacturing. It begins by elucidating the critical steps in upstream vector cGMP production
platforms, including Transient Transformation (TT), Baculovirus Expression (rBV/Sf9), Herpes Simplex
Expression (HSV/BHK), and Producer Cell Lines (PCL). It continues by focusing on CMC strategies and precursor materials for each production platform. Purification strategies, AAV vector product characterization, analytical testing for safety and quality, viral vector manufacturing facility design, and regulatory considerations for AAV vectors are considered in detail. Take the next step and learn the highly technical aspects of AAV gene therapy manufacturing. Enroll today!

Section 1: AAV Overview

Provides an overview of the properties of the naturally occurring Adeno-associated virus including its origin and characteristics.


Section 2: AAV Vectors

Describes how the adeno-associated virus is utilized as an AAV vector. Includes a discussion of the general characteristics and composition of AAV viral vectors. Ends with a survey of genes that can be transfected using these vectors.

Section 3: AAV Manufacturing and Controls

Discusses AAV Manufacturing and control including an introduction to manufacturing, the four primary platforms for AAV production, the characterization of AAV manufacturing products, and the regulation of AAV production.

Five Takeaways


  1. Explain how the structure and functions of the AAV virus influence its tissue tropisms.
  2. Analyze the challenges faced in manufacturing recombinant AAV gene therapy vectors.
  3. Identify the strengths and weaknesses of the different AAV vector production platforms Transient Transformation (TT), Baculovirus Expression (rBV/Sf9), Herpes Simplex Expression (HSV/BHK), and Producer Cell Lines (PCL).
  4. Examine downstream bioprocessing strategies, including CMC strategies and precursor materials, for each production platform (TT, rBV/Sf9, HSV/BHK, PCL).
  5. Assess the key considerations in viral vector manufacturing facility design and regulatory requirements for AAV vectors, ensuring compliance with safety, identity, potency, quality, and
    purity testing.

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