Keynote Presentation: Gene Therapy Manufacturing with Live Q&A

The field of gene therapy has enabled the treatment and potential cures for serious, monogenetic diseases.  This form of advanced therapy relies on the delivery of the “correct” copy of a gene to a patient’s cells, and often utilizes a viral vector to achieve this goal.  This presentation will give an overview of the history of gene therapy products as well as insight into the current state of the market and the size of the pipeline.  In addition, a broad overview of the cGMP manufacturing process for adeno associated virus (AAV)-based gene therapies will be covered, including unit operations for production, purification, and formulation.  Finally, other relevant topics such as targeted drug delivery, navigating the regulatory process, and the price point of gene therapy products will also be covered.  

Learning Objectives:

1. Define what gene therapy products are.

2. Describe different types of vectors for gene therapy.

3. Outline a general process for AAV manufacturing.