Fundamental Constriction Points in Emerging Therapies, High-Titre Vector-Producing Cells

In this webcast, panel speakers will discuss challenges. From both a drug development and also clinical perspective. We will learn why analytical characterization of material is important at every step to making sure the critical quality attributes (CQA) stay within a threshold, within specifications and guidance, are identify their potential Chemistry, Manufacturing and Controls (CMC) issues, which need to be tackled during the early manufacturing process development.

Register Free: https://www.pharmtech.com/pt_w/high-titre

Event Overview:

In this webinar learn that manufacturing for viral vectors complexity requires components that are not needed in stable, recombinant protein–expressing production systems. Examples are lipid- or polymer-based transfection reagents and lysis buffers. Another added obstacle, overall yields of functional capsids containing the desired genetic payload is most often lower than required, creating calls to move to higher scales of production to compensate. Furthermore, transient transfection is frequently employed constituting a single-run bespoke cell line for each manufacturing lane. A more robust, scalable, and higher density suite of methods is therefore highly desirable.

Key Learning Objectives

• Achieving high-yield manufacturing of plasmids that contain difficult genetic payloads (gene-of-interest size, base composition, toxicity, use of selection markers)
• Relative importance of these challenges when pursuing large plasmid sizes, for example, leakey promoters that lead to pre-mature protein expression upstream or losses during alkaline lysis. Can we discount for repeated sequences such as ITRs and polyA tails that are more prone to deletion and recombination events?
• Are there successful ways to navigate these by balancing buffer volumes, buffer contact time, reducing sheer, using well characterized pHelper and pREPCAP plasmids
• How automation might decrease manual labor, however integration isn’t easy – problems exist in communication with reactors, analyzers and pre-existing software.
• Pros and cons for industry to develop either a series of dedicated fit-for-one purpose optimized platforms, versus attempts at a “flexible” platform that can manage a wide ranch of vector types which might then be “standardized” industry-wide.

Who Should Attend

• Advanced therapy medicinal products scientists
• Cell and gene therapy manufacturers
• Regulatory and government relations personnel
• Viral vector manufacturers
• Oncologists and immunologists
• Potency assay researchers
• CAR-T and blood disorder physicians

Speakers:

Dr. Nicole Faust
General Manager
Cell Line Development at Cytiva

Dr. Nicole Faust has more than 20 years of management experience in the biotechnology sector. She joined Cytiva in 2022 through Cytiva’s acquisition of CEVEC. A member of the CEVEC team since 2011, she held several roles including CSO and CEO. She was instrumental in developing CEVEC´s unique ELEVECTA technology for scalable AAV production. Before joining CEVEC, she held leadership positions with Lonza and Taconic Biosciences. Her scientific background is in stem cell biology and she holds a PhD in cell and molecular biology from University of Freiburg and an MBA from Educatis University, Switzerland. Dr. Nicole Faust is well connected in the cell and gene therapy community, is a member of the American Society for Gene and Cell Therapy and currently Chair of the Gene Therapy Advisory Committee of the Alliance for Regenerative Medicine (ARM).

Dr. Vibha Jawa
Executive Director
Head of Biotherapeutics Bioanalysis in Nonclinical Disposition and Bioanalysis (NDB) OrganizationBristol
Myers Squibb

Dr. Vibha Jawa is currently an Executive Director in the Nonclinical Disposition and Bioanalysis for Biotherapeutics group at Bristol Meyers Squibb. Previously, she led the Predictive and Clinical Immunogenicity group Director of Preclinical Development at Merck Research Labs where she was responsible for developing a strategy and provide oversight and management of scientific programs for discovery, development and optimization of biologics and vaccines. Her current research interests include evaluating immune response biomarkers for early drug development and efficacy, immunogenicity prediction using in silico, in vitro and in vivo technologies, modeling impact of immunogenicity on PK and PD and their application to a systems based approach, antigen processing and presentation and the role of T cells in immune response to drug products

Benjamin McLeod
Technical Lead
Virica Biotech

Benjamin serves as Technical Lead with Virica Biotech, a yield-enhancing solution for viral vector and cell therapy production. He is trained in cell biology, biotechnology, and viral vector production. You can find him on LinkedIn, where he regularly posts industry insights and curates papers on process development in CGT.

Register Free: https://www.pharmtech.com/pt_w/high-titre

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