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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Optimizing process development to simplify manufacturing scale up of therapeutic viral vectors

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With the progress in developing new viral vector systems guided by safety, specificity and potency considerations, several gene and cell-based therapies are now close to being clinically approved and commercially available to treat genetic diseases. These viral vectors systems are based on the production of mainly adeno-associated viruses (AAV) and lentiviruses by transient transfection of human embryonic kidney (HEK)-293 derived producer cell lines.

One of the bottlenecks that needs to be addressed is optimization of production during Upstream Process Development to simplify manufacturing scale-up of therapeutic viral vectors to support advanced clinical trials and commercialization. This is why, virus vector production using the right transient transfection approach and combined cell culture system is crucial to achieve process performance, while complying with Quality requirements for process development and clinical-grade manufacturing. With the available quality grades of PEI, PEIpro®, PEIpro®-HQ and recently launched PEIpro®-GMP, Polyplus-transfection® offers a complete and reliable PEI transfection method that guarantees direct scalability and seamless transition from process development up to large-scale clinical-grade viral vector manufacturing.

During this On Demand you will hear from experts in transfection, advanced culture systems and gene therapy viral vector manufacturing to optimize your process development and simplify your manufacturing scale-up.

Chris Lorenz, Senior Director, Process Sciences & Manufacturing, Audentes Therapeutics, Inc.

Chris Lorenz received his BS and MS in Chemical Engineering from Stanford University. He began his career at Genentech in their Early Stage Cell Culture department, developing new processes for Genentech’s pipeline of programs moving from Research into Development. He transitioned into a PD role focused on new technology evaluation with an emphasis on single-use technologies, and later joined their Manufacturing Science & Technology (MSAT) organization. In 2010, Chris joined Novartis Diagnostics on their Engineering team, and from there moved into Manufacturing where he managed various functions within Novartis’s immunodiagnostics operations (which was acquired by Grifols in 2014). Chris joinedAudentes Therapeutics in early 2016, where he was brought in to start up their internal manufacturing operations from scratch. In late 2016, Audentes began GMP manufacturing for their pipeline of AAV-based gene therapy programs. He currently leads their AAV Process Development, Process Engineering, and Manufacturing functions.

Dr Valérie Kedinger, GMP & Analytical Subcontractor Manager, Polyplus-transfection

Valerie Kedinger holds a PhD in Molecular and Cellular Biology from Strasbourg University. After a three-year postdoctoral fellowship in cancer research at McGill University (Montreal, Canada), she joined Polyplus-transfection® in 2011 as the in vivo R&D project leader to drive the development of in vivo nucleic acid delivery approaches in cancer treatment. Valérie went on to serve as in vivo and Bioproduction Application Specialist to provide scientific guidance to preclinical and clinical-stage Biotechs and Pharmas. Since 2018, Valerie is the GMP and Analytical Subcontractor Manager at Polyplus-transfection.

Dr Rachel Legmann, Senior Manager of Cell Culture Process Development Lab, Pall Life Sciences

Dr. Rachel Legmann, PhD is a Senior Manager, leading the cell culture PDS-WB-US laboratory within the Single Use Technology (SUT) Biotech business unit at Pall Life Sciences. At Pall Biotech Rachel is focusing on technology transfer of various viral vectors providing manufacturing support activities combining biological and engineering aspects and maximizing process efficiencies and product quality for viral vector manufacturing production. She completed her Ph.D. in Food Engineering and Biotechnology at the Technion-Israel Institute of Technology, Israel. In 1996 she joined the Microbiology & Molecular Genetics department at Harvard Medical School and was part of the development team for large scale manufacturing processes for new recombinant protein vaccines. In 2002 Rachel joined the pre-clinical contract research organization, SBH Sciences as Principal scientist. Her contributions included optimizing existing cell culture processes for better product yield and quality as well as expanded new products line and services. Rachel joined Seahorse Biosciences in 2007 as an associate director. As a member of the management team her responsibilities included designing and optimizing upstream and downstream processes for a new high throughput cell culture automated micro-bioreactor automated platform, and developing multiple cell based metabolic assays and kits for the Extracellular-Flux cell metabolism analyzer. In 2013 she joined Goodwin Biotechnology, a Contract Manufacturing Organization (CMO), as an upstream process development manager. Rachel joined Pall in 2014 and currently serves as a process development services senior lab manager in charge of upstream and downstream process development, scale-up, continuous bioprocessing and manufacturing support activities for gene therapy and biologic market.

Kathy Webb, Senior Director GMP Operations, Paragon Bioservices, Inc

Kathy Webb, MSChE, Senior Director GMP Upstream Manufacturing, is responsible for the upstream manufacturing operations at Paragon Bioservices and has experience with a variety of production processes utilizing stable cell lines, transient expression, baculoviral expression vectors, and microbial fermentation. Kathy has over 20 years of experience working in a contract development and manufacturing organization and has had evolving roles and responsibilities in process development, technical support and the manufacture of biologics. She has a M.S. in Chemical Engineering from the University of Illinois at Urbana-Champaign and a B.S. in Chemical Engineering from the University of Pittsburgh.

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