Future-proofing gene therapy: scalable AAV solutions and non-viral innovations

Thursday 08:00 PDT / 11:00 EDT / 16:00 BST / 17:00 CEST

Sponsor

Future-proofing gene therapy: scalable AAV solutions and non-viral innovations

As gene therapy moves toward broader commercialization, the pressure to streamline production and ensure consistent, high-quality output has never been greater. Here, we explore strategies to develop simplified, scalable state-of-the-art platforms designed to optimize AAV yields and process efficiency. Whether you're facing bottlenecks in upstream development, struggling with analytical limitations, or exploring safer, more efficient alternatives to viral vectors, this session delivers practical insights grounded in real-world data and case studies.

This webinar will walk you through novel approaches to AAV production using stable producer cell lines achieving a titer higher than 1E12 vg/mL with > 30% full capsid at harvest. We explore innovative robust analytics for comprehensive product characterization looking at AAV genomic integrity and its correlation with orthogonal analytical methods. We will also introduce emerging non-viral delivery systems including extracellular vesicles (exosomes) which counter concerns around viral vector safety and toxicity. Here, we elaborate on vector and cell line development to build a platform-based approach to manufacturing.

Attendees will learn about:

Actionable strategies for scale-up: see case study data on how to implement simplified, state-of-the-art AAV production platforms that meet the growing demands of commercial gene therapy
Smarter analytics for better outcomes: explore innovative approaches to overcome current limitations in AAV analytics and achieve comprehensive product characterization
Future-ready alternatives: be introduced to emerging non-viral gene delivery platforms like exosomes, which offer promising solutions to address safety and toxicity concerns

Vijetha Bhat

Manager, Research & Development at Lonza Licensing

Vijetha Bhat is a technology expert working within Licensing in Lonza’s Specialized Modalities division. She has 15 years of experience in the biopharma industry. Vijetha started her scientific career at Lonza in 2017 and has since contributed to a variety of projects focusing on developing production platforms and analytics for the cell and gene therapy space. She currently supports with providing in-depth technical direction to users working with Lonza’s expression platforms.