Avoiding scale-up pitfalls: upstream process development strategies for AAV manufacturing

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The gene therapy field is on the cusp of widespread use of recombinant AAV for addressing previously untreatable and incurable diseases. However, the manufacturing of recombinant AAV therapeutics has historically been inefficient. It is not uncommon for bioreactors at the hundred-liter scale to yield a limited number of doses suitable for patients. Additionally, processes that scale effectively from shake flasks to benchtop bioreactors often encounter challenges when transitioning to larger scales, such as 100-L bioreactors and beyond.

Nevertheless, these challenges can be mitigated through meticulous and strategic process development. A thorough understanding of cell behavior and productivity within each type of bioreactor is crucial for establishing a reliable baseline level setting. Further, the scalability of the transfection step must be evaluated early in the process development—particularly regarding the logistics of plasmid DNA and reagent mixing, as well as the timing of aseptic transfection mixture addition into the bioreactor.

In this webinar, the speakers will present a detailed case study on optimizing AAV5 production, covering aspects such as plasmid ratio optimization, bench-scale experimentation, and the successful scale-up to a 200-L bioreactor for full production. The speakers will also introduce a novel transfection technology that enables large-volume bioreactor runs for viral vector production, that reducing the risk of process failure.

Attend this webinar to:

• Explore the challenges that are encountered when scaling-up AAV production
• Gain insights from a case study on upstream process development for successful AAV production in a 200-L bioreactor
• Delve into transfection reagents which improve AAV yields and streamline workflows

Sandy Tseng

Field Application Scientist at Mirus Bio LLC

Sandy Tseng has been a key member of the Technical Support team at Mirus Bio since 2019, where she collaborates closely with scientists to troubleshoot and optimize their gene delivery experiments and processes. Prior to joining Mirus Bio, Sandy completed her doctoral research on the regulation of eukaryotic gene expression at the University of Wisconsin-Madison. Her expertise bridges emerging biotechnology with practical applications in biomanufacturing.

Hugh Murray

Process Development Senior Scientist at MilliporeSigma

Hugh is a dynamic biotech scientist with over a decade of process develop experience designing, optimizing and scaling-up mammalian suspension processes for biologic and gene therapy production. He holds a B.S. in Genetics from the University of California, Irvine. He is a Senior Scientist in the Upstream Process Development team at MilliporeSigma’s Viral Vector CDMO site in Carlsbad, California.

In this role, Hugh has led the sites efforts to develop an upstream platform process for AAV viral vector production. Hugh is a specialist in Design of Experiment (DoE) methodology and predictive modeling, and has coupled this skillset with the use of automated robotic micro-bioreactor and liquid handler platforms to create a development workflow that can take a client’s “gene and a dream” into clinical phase manufacturing rapidly and efficiently. Hugh also has extensive experience successfully scaling bench-scale processes into large scale single use bioreactors (SUB) with a high degree of comparability between scales.

Prior to working in the viral vector space, Hugh worked in monoclonal antibody development at Tanvex BioPharma Inc. There he worked on early and late-stage process development, process characterization and validation, and authored BLA CMC sections for commercial approval of biosimilars. He also managed the MSAT team during phase III clinical material production of multiple biologics.

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SPEAKERS

Sandy Tseng

Field Application Scientist at Mirus Bio LLC

Hugh Murray

Process Development Senior Scientist at MilliporeSigma

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