Addressing the Challenges of Purification & Quality Control in Gene Therapy
AbstractRecombinant Adeno Associated Virus (rAAV) vectors are one of the most promising mechanisms for therapeutic gene delivery. However, most industrial cell lines exhibit inefficient gene packaging which results in a heterogenous population of vectors composed of empty, partially-loaded, and fully-loaded species. Purifying, identifying, and quantifying these different species is vital from a production and quality control standpoint, since improperly loaded viral capsids do not produce the desired therapeutic effect but may still elicit an unintended immune response.
In this Beckman Coulter Life Sciences webinar, our panelists will focus on several pertinent topics in rAAV process development including current challenges, technologies for overcoming quality control hurdles, best practices for downstream processing, as well as discuss the current trends and future outlook of this rapidly growing therapeutic frontier.
- Understand the challenges of differing serotypes in rAAV production and quality control
- Understand the commonly used characterization techniques for AAV capsids
- Understand the basics of Analytical Ultracentrifugation (AUC) and how it is used to characterize empty, full, and intermediate AAV capsids
- Learn about best practices in Density Gradient Ultracentrifugation (DGUC) to achieve highest purity in gene therapy products
- Dr. Akash Bhattacharya, Senior Applications Engineer, Beckman Coulter Life Sciences
- Dr. Audrey Chang, Vice President of Quality Control and Analytical Service, Vigene Biosciences
- Leisha Kopp, Applications Scientist, Mirus Bio
- Dr. Klaus Richter, Group Leader AUC, Coriolis Pharma
- Dr. Shawn Sternisha | Senior Field Applications Scientist, Beckman Coulter Life Sciences