Thermo Fisher Launches System to Scale Gene Therapy Workflows

Thermo Fisher Scientific recently launched the integrated Gibco AAV-MAX Helper Free AAV Production System to enable scalable gene therapy workflows.

The solution aims to simplify AAV vector production workflow, increase productivity, and boost cost efficiency by delivering high viral titers using Viral Production Cells 2.0, a clonally documented, 293F-derived mammalian cell line. 

AAV is vital for gene therapy production, as over 1,300 unique gene therapy products are currently under development and almost half are reliant on AAV. Scaling production decreases costs and boosts the overall process from research to commercialization.

"AAV has an efficacy and safety profile that makes it an ideal therapeutic vector and one of the go-to biological delivery methods for the latest gene therapy breakthroughs,” Amy Butler, president of biosciences at Thermo Fisher, said in the announcement. 

"The new AAV production system is part of our end-to-end workflow solutions designed to meet growing demand for cost-effective, scalable viral vector production. Having access to products and services designed specifically for cell and gene therapy research, with line of sight to clinical manufacturing, empowers our biotech and biopharmaceutical partners,” Butler continued. 

Thermo Fisher developed the AAV-MAX system to scale from shake flasks to bioreactors. 

The system can save viral vector research and developers 25 percent on plasmid DNA usage and 50 percent on production costs compared to polyethyleneimine (PEI)-based mammalian production ecosystems, a Thermo Fisher spokesperson said. 

Developers can use the AAV-MAX system and Thermo Fisher’s expanded gene therapy portfolio to increase commercial production and help deliver a wide variety of therapies to patients. 

A 2020 PhRMA report found that there are 362 investigational cell and gene therapies in clinical development last year, a 20 percent increase from 2018. 

The growing interest in gene therapy has led to a need for more cost-effective and scalable vector manufacturing platforms to deliver therapies to patients. Generally, developing AAV vectors includes plasmid development and production, cell expansion, plasmid transfection, viral vector production, purification, and fill and finish.
This year, companies such as Biogen and Capsigen and CRISPR Therapeutics and Capsida partnered to develop gene therapies for chronic diseases by leveraging AAV vectors.