AAV process development & scale-up

The adeno-associated viral (AAV) vector-driven gene therapy field continues to strive to overcome the obstacles to its successful industrialization and migration to indications with larger patient populations. Diverse priorities vie for attention and budget, including enhancing the scalability and productivity of upstream processes, improving downstream process recovery rates, and harnessing the power of bioanalytical innovation. Here, a panel of three AAV vector processing and chemistry, manufacturing and control (CMC) experts share their insights and advice relating to several key questions facing the field today, including:

• What are the chief obstacles to streamlined, cost-effective and robust scale-up  of AAV vector processes? What have been the important recent advances in AAV process intensification – for example, in terms of enabling viral vector process intensification at smaller scales
• Staying with analytics, where have we seen the greatest progress of late – for instance, in enabling the identification and monitoring of critical quality attributes (CQAs)?
• Looking to the future, what is the likely direction of future CMC-related regulatory evolution in the AAV space, and where will process and analytical tool innovation occur as a consequence?
• Finally, what are some key process- and product development–related considerations when preparing to meet the commercial challenges of vector manufacture?

Catherine Cancian is Chief Technology Officer at ATAMYO Therapeutics. She has worked in the Biologics CMC/ Process Development/ Regulatory Compliance for clinical and commercialization phases for the past 25 years, focused since 2015 on the viral-vector gene therapies. Prior to joining Atamyo, she was Vice President of the Pharmaceutical Operations at GenSight Biologics where she led the strategy for CMC and Supply Chain readiness to prepare for early commercial phase. Before that, she was industrial Project Director for the setup of a new Gene & Cell Therapy Manufacturing facility at YposKesi. She started at Sanofi Pasteur where she served during almost 18 years at various leadership positions in vaccines Manufacturing, Process Sciences & Technology, Quality, and CMC Project Management leading multifunctional teams for Process Transfer, manufacturing readiness and filing for regulatory submissions.

Karen Doucette currently serves as Cell and Gene Therapy (CGT) Navigator at Charles River Laboratories. Karen plays a critical role in partnering with clients to map the development journey from discovery through commercial manufacturing, with a focus on process development guidance to avoid common, time-consuming pitfalls. Karen has over 20 years of experience with biopharmaceutical CROs and CDMOs, spanning pre-clinical and clinical research, development and manufacturing. She has been instrumental in collaboratively driving expansion of CGT service portfolios and aligning research innovation with customer need. Karen received her BSc in Animal Science from the University of Delaware and completed her MBA at Goldey-Beacom College.

Reinout Hesselink is a freelance consultant for senior level CMC/QA roles in cell and gene therapy His professional focus emphasizes on the CMC and development of cell and gene therapies and their production process from early clinical up to commercial products. Reinout has a proven track record as a general manager and experience in preparation for clinical and commercial supply of cell and gene therapies. Roles in companies as Lonza, BioNTech, ExCellThera, Cimaas and Pan Cancer T as well as consulting for many others including big pharma has given him an in-depth understanding of the clinical and commercial requirements of developing, manufacturing and testing cell based therapies.

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