3 questions with…
Global Senior Principal Scientist,
What are the biggest challenges in the advanced therapy industry?
I believe that Chemistry, Manufacturing and Controls (CMC) is one of the biggest challenges in the industry. Within the advanced therapy categories of allogeneic cell therapies, autologous cell & gene therapies and viral vector gene therapies, the CMC challenges are all different. For allogeneic cell therapies, I think that tissue sourcing is the biggest challenge as the foundation of the therapy is the quality of the initial donor tissue. Ensuring quality donor tissue whilst complying with all the relevant ethic principles, laws and regulatory rules is highly challenging. In the case of autologous cell & gene therapies, I think the biggest challenges are scalability and analytical testing, given the nature of the therapies where it is one GMP batch per patient it is inherently difficult to scale. And with the analytics it is very difficult due to the inherent variability of the product and there is limited understanding of the analytical quality of this type of product and how it links to clinical efficacy. Finally for viral vector gene therapies, I believe the biggest challenges are the scalability of the manufacturing processes and effective analytics to understand the quality of these products. Given the plasmid starting materials required for most viral vector processes, this limits the scale you can achieve with this type of product.
What advanced therapy innovations are you most excited about?
There are so many innovations ongoing in the advanced therapy space but one area that excites me is the improved automation of cell therapy manufacturing processes, the consistency and quality of cell therapy products represent significant manufacturing challenges. The release of the updated Cocoon Platform in 2022 by Implementing Magnetic Cell Selection allows the automation of cell selection and separation increasing overall process efficiency, minimising user input variability and allowing for easier scale-up.
What are your predictions for the next 5 years for the Advanced Therapy Industry?
In the next 5 years I predict there will be significantly more advanced therapies approved for commercial use in all categories; allogeneic cell therapies, autologous cell & gene therapies and viral vector gene therapies. But I believe that allogeneic cell therapies have the most potential to become more ubiquitous across the pharmaceutical industry because of its advantages over autologous cell & gene therapies. I can see alternative viral vectors being used that have lower immunogenicity and higher efficacy than the ones being used at the moment. Improvements in the scalability of manufacturing processes and analytics will enable advanced therapies to reach new markets and expand beyond rare diseases to make them available to treat diseases with large patient populations. I hope to see the cost of good decrease as it is one of the major drivers for their high market price point.