Big Data Strategies for Cell Therapy Manufacturing
The quest to retrieve, analyze, and leverage that data has become the new gold rush in life sciences. This presentation will discuss the role of big data in cell therapy process development, real time analytics and commercial scale manufacturing.
RecordedSep 17 201976 mins
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Karen Zink McCullough, Owner, Principal Consultant at MMI Associates
Presented by Karen Zink McCullough, Owner, Principal Consultant at MMI Associates
The Bacterial Endotoxins Test is not a test for pyrogens (fever causing agents). It is not a test for the presence of Gram negative bacteria. Based on extensive comparisons of endotoxins activity in the BET and fever rabbits executed in the 1970s and 1980s, we may say that the BET is a test for levels of Gram negative bacterial endotoxins activity that may be predictive of a fever in mammals.
The various methodologies that are described in the harmonized in the harmonized chapter use the primary Reference Endotoxin Standard (RSE) or secondary Control Standard Endotoxin (CSE) to prepare assay standards, perform suitability testing as instructed in the chapter and inoculate positive product controls. These standards are relatively pure preparations of lipopolysaccharide. However, the utility of the compendial assay for non-compendial applications is limited. Our discussion will focus on the compendial uses of these BET assays.
Dr Friedrich von Wintzingerode, QC Lead iNeST Project (individualized Neoantigen Specific Therapy) at Roche
FULL TITLE: A Comprehensive Approach to Assess the Impact of Microbial Impurities on Patient Safety and Product Quality of Biologics
Presented by Dr Friedrich von Wintzingerode, QC Lead iNeST Project (individualized Neoantigen Specific Therapy) at Roche
Large-scale Production of Biologics is susceptible to microbial contamination because many manufacturing steps occur under non-sterile conditions in aqueous systems at ambient temperature or 2-8 °C under substantially neutral pH conditions. Regardless of where in the Drug Substance (DS) manufacture (manufacture of the Active Pharmaceutical Ingredient), or Drug Product (DP) manufacture (manufacture of the Final Drug, e.g. formulated mAbs filled in vials or syringes) they occur, microbial contaminations can have a significant impact on product quality and patient safety. Even after bioburden removal by 0.2 µm filtration subcellular microbial components like toxins, lipopeptide/lipoproteins, flagellin, bacterial and fungal DNA, cell wall polysaccharides, extracellular proteases or endoglycosidases remain in the product. Those microbial components potentially lead to toxic, allergic or inflammatory responses in humans or product degradation or modification. The Case-by-Case Assessment of Bioburden (CCAB) approach described here enables a comprehensive assessment of these risks.
Dr Christine P. Chan, Ph.D. Director in Global Manufacturing Science & Technology at Sanofi
Presented by Dr Christine P. Chan, Ph.D. Director in Global Manufacturing Science & Technology at Sanofi
Bioprocess changes can impact quality attributes of biologics and may affect efficacy and safety of the product. During development and throughout the product lifecycle, when process improvements are implemented, it is essential to gather sufficient data to support the conclusion that product safety and efficacy has not been adversely affected. This demonstration exercise requires careful planning of the comparability studies and is based on the background knowledge of protein structure, biological function, and clinical attribute profiles of the product accumulated during development.
In this webinar, I will discuss strategies and considerations for analytical characterization of protein structure and function which forms the foundation of the comparability demonstration.
Presented by Dr. Jincai Li, Vice President of WuXi Biologics
With the rapid growth of the biotherapeutics industry, the need and challenge for high quality cost efficient production have been increasing as well. At the same time, the number of approved biologics products are also steadily increasing, and more and more products are being developed by small to mid-size biotech companies, with product market size that varies greatly and therefore leading to varying production scale needs. The presentation will talk about the paradigm shifts in today’s facility design and operations, with the multi-purpose facility and smaller, the modular facility being favoured by many companies. In addition, rapid adoption of disposable technology has enabled faster and lower cost facility design & start-up. With the modular, disposable technologies, the unique “scale-out” approach has the advantage of providing the highest flexibility to customers while simultaneously lowering tech transfer and scale-up risks. The presentation will also cover the continuous bioprocessing concept and share WuXi’s efforts on this area
Presented by Dhaval Tapiawala, Principal Scientist at Pfizer
• Understanding extractables and leachables for better adoption of single use systems
• Ensuring safety of drug through determining the level of leachables throughout product life cycle
• Case Study of implementation of standardized testing protocol
Michelle Raikes, M.S., Scientist IV and Dr. Fredrik Nordstrom, Sr Research Fellow at Boehringer Ingelheim
Full Title: Quantification and Control of Amorphous contents by Raman, Application and Case Studies in Pharmaceutical Processing
Presented by Michelle Raikes, M.S., Scientist IV and Dr. Fredrik Nordstrom, Sr Research Fellow at Boehringer Ingelheim
Amorphous regions in crystalline material can have significant impact on bioavailability, processability and stability. Conversely, crystalline material can act as nuclei for recrystallization and resulting in instability of amorphous systems. Raman spectroscopy is sensitive to polymorphic differences and amorphous content in pharmaceutical materials. This make Raman spectroscopy a powerful process analytical technology (PAT), when combined with multivariate modelling, to control the amorphous content in a pharmaceutical process. We will present pharmaceutical case studies showing process-induced amorphization of crystalline drugs across the DS and DP processing steps. In addition, we will compare the kinetics of crystallization of amorphous material when stored at various relative humidity and temperature conditions. The results clearly demonstrate that Raman spectroscopic techniques combined with multivariate methods is a powerful and effective tool for quantitation of amorphous content in crystalline material with applications ranging from API isolation, milling and to multiple unit operations in DP manufacturing. The information generated was critical to determine the root-causes and outline appropriate mitigation measures
Lars Hovmand-Lyster, Senior Engineering Specialist in Novo Nordisk Global Project Office (GPO)
Presented by Lars Hovmand-Lyster, Senior Engineering Specialist in Novo Nordisk Global Project Office (GPO) Engineering Management Department
• Build the roadmap to biopharmaceutical manufacturing and their partners in developing, assessing and verifying unit operations leading to an appropriate facility design that is “right classified”
• Hear first-hand about this industry-developed document to understand the tools, steps and documents required to adequately risk assess and qualify modern facilities that make use of modular, closed technology
• Learn how following a methodology results in significant gains in reducing the time to deliver the facilities and CAPEX and OPEX costs
Ken Wong of Sanofi Pasteur, Desmond G. Hunt, of USP and James Hathcock of Pall Biotech
Full Title:Utility of Generating Data: Drug Manufacture’s Perspective: Will USP permit such format?
Presented by Ken Wong, Deputy Director at Sanofi Pasteur
This talk will focus on an overall application of USP starting from risk assessment to qualification of disposable manufacturing systems based on USP data set. All the key principles with examples where these principles need to be satisfied before one can apply the USP data for disposable manufacturing system qualification will be discussed and illustrated. Lastly, different qualification approaches will also be presented to provide broader understanding.
Followed by a presentation by Desmond G. Hunt, Principal Liaison at United States Pharmacopeia
Dr. Desmond G. Hunt has been with USP since 2005 and holds the position of Principle Scientific Liaison in the Compendial Science Group-General Chapters. He is the scientific liaison to the Packaging and Distribution and Dosage Forms Expert Committees, where he works to develop and revise USP Standards. He has authored many publications and peer-reviewed articles and is a frequent speaker and instructor on topics related to pharmaceutical packaging, particulate matter in parenteral and ophthalmic dosage forms and good storage and transportation practices. He participates on several industry Working Groups and Technical Committees related to his areas of expertise. Dr. Hunt obtained his M.S. and Ph.D. from the University of Texas at Austin and prior to joining USP, was a Research Fellow at the National Institutes of Health, Bethesda, MD, USA
And Followed by an Industry Perspective presented by James Hathcock, PhD, Sr Director, Regulatory and Validation Strategy at Pall Biotech
Francis Verhoeye, Global Pilot Operations Leader at Zoetis Inc.
FULL TITLE: Single-Use Bioprocess Platform for Veterinary Vaccine and Biopharmaceutical Pilot-Scale Production
Presented by Francis Verhoeye, Global Pilot Operations Leader at Zoetis Inc.
•Understand the implementation journey taken to accelerate veterinary biological product development
•Identify the challenges of integrating vaccine and biopharmaceutical processes
•Learn about the single use qualification and implementation process in a multi-product facility
Full Title:Advanced Raman Spectroscopy Beyond In-Process Glucose Control of Commercial Cell Culture Processes
Presented by Dan Hill, Manufacturing Scientist at Biogen
The use of in-line Raman spectroscopy as an in-line process analytical technology (PAT), in the biotechnology industry, has matured over the past decade from a technology with promise to a standard tool for real-time, continuous monitoring of cell culture processes. There is an ever-growing body of work demonstrating successful application from metabolite and product quality monitoring to process control in both process development and GMP manufacturing environments. This is due, in part, to advances in technology robustness, user- and integration-interfaces, improved sensitivity, and fluorescence rejection. Organizations are now faced with the difficult task of best leveraging these tools and extracting the most value from them. Thus, defining and executing Raman program strategy is critical to the technology’s long-term success and sustainability.
This presentation will describe Biogen’s past efforts and strategic direction as it relates to in-line Raman spectroscopy for cell culture operations and how we intend to leverage its capability beyond metabolite monitoring and glucose feedback control to become an essential element of our Advanced Process Control efforts.
Danny Chou, President and Founder at Compassion BioSolution, and Mark Bumiller, Technology Manager at Particle Sizing Systems
Presented by Danny Chou, President and Founder at Compassion BioSolution, LLC
Followed by an industry perspective presented by Mark Bumiller, Technology Manager at Particle Sizing Systems, an Entegris Company
In the past decade, we have witnessed the arrival of a large number of analytical technologies that are useful for characterizing sub-visible particles in protein therapeutics. Even with the diverse tools that are available today, there are still important gaps that have not been filled but yet have a significant role in our ability to fully analyze particles for either product characterization or formulation development purpose. The goal of this presentation is to highlight some of these gaps and share the opportunities that may be captured by new tools that are on the horizon. Finally, the speaker will elaborate on how simultaneous monitoring of sub-micron and micron-sized particles can assist biopharmaceutical formulation development and help fulfill current and future regulatory requirements.”
Lawrence De Belder, Senior Principal Engineer at j&J and Bob Lenich, Director of Life Sciences Business at Emerson
Presented by Lawrence De Belder, Senior Principal Engineer Continuous Manufacturing at Johnson and Johnson
Followed by an Industry Perspective presented by Bob Lenich, Director of Life Sciences Business at Emerson
The counter is at Six today: Orkambi, Symdeko, Trikafta (Vertex); Prezista (Johnson and Johnson); Verzenio (Eli Lilly); Daurismo (Pfizer) –are all approved by the FDA – Johnson and Johnson’s Tramacet for the Japanese market not included. Several large Pharma companies have chosen the path of Continuous Manufacturing, and announced they will use this platform as their default for development and consequently – commercial manufacturing of their Oral Solid dose drug product pipeline.
During this webinar, the author will explain the consequences of this decision, and different strategies to handle a pipeline that can be highly dynamic. Different techniques will be discussed that can be used to bring products with bad flow properties into a continuous process. Impact of different equipment in development, clinical and commercial environment will be detailed out, and mitigations will be proposed to overcome these differences. All will be placed in front of a background of regulatory requirements, changing market demand, and evolving strategies of equipment vendors.
Andrew Teasdale, Senior Principal Scientist at AstraZeneca and Piet Christieans, Phd,Scientific Director at Nelson Labs
FULL TITLE: Assessing the Risk of Interaction Between Extractables and Leachable and Therapeutic Proteins
Presented by Andrew Teasdale, Senior Principal Scientist Impurity management and External Advocacy at AstraZeneca
Biologics and Peptides often present their own challenges in respect to leachables. They are typically administered parenterally and therefore there is a high risk of interaction between dosage form and packaging / administration system. They are often low dose and sensitive to structural modifications, this can impact Safety (immunogenicity), Quality (instability, aggregation) and Efficacy (loss of potency). This webinar will examine the potential risk factors through case study examples including reactive leachables, the impact of sterilisation and adverse events and their route cause. It will conclude by looking at opportunities to look predictively at potential issues and how this can be incorporated into product design
Followed by Piet Christieans, Phd,Scientific Director at Nelson Labs
Mikkel Nissum, Vaccine R&D Quality Site Lead at GSK Vaccines
Presented by Mikkel Nissum, Vaccine R&D Quality Site Lead at GSK Vaccines
Only clean drinking water rivals vaccination in its ability to save lives. Yet, development and manufacturing of vaccines remain challenging. Vaccines comprise a heterogeneous variety of targets. Each target requires a dedicated development and manufacturing process adding to the timelines for getting new vaccines to the patient and to ensure a reliable supply. For the downstream processing part, often the process is composed of an optimized set of up to three chromatographic steps to provide the final desired quality of the target. On top of the long development time required to develop such a multi-step process, at times, the end result may not be satisfactory, in particular regarding purity of the target.
In order to overcome this downstream processing challenge, we embarked on establishing a purification platform based on affinity purification. The main advantages of such a platform for vaccine manufacturing would be:
1)By applying a positive selection principle for chromatography, the number of chromatographic steps may be reduced to just one step
2)Establishing a platform would require minimal process changes from one target to another
3)Downstream processing would become predicable in terms of development time and costs
The journey of establishing the affinity purification platform focusing on main challenges and key results will be presented in the Webinar.
Moritz von Stosch, PhD, Senior Manager, Technical R&D, GlaxoSmithKline Vaccines
Machine Learning is believed to be a game changer for industry, especially by big Pharma as reflected by significant investments. Starting from an introduction to machine learning, this contribution outlines the current machine learning status for process development with examples, provides future directions towards more global high-value machine learning centric development, concepts to engage people in the digital evolution and ideas for machine learning centric business models.
Presented by Moritz von Stosch, PhD, Senior Manager, Technical R&D, GlaxoSmithKline Vaccines
Moritz von Stosch works as Senior Manager at Technical R&D of GSK Biologicals, Belgium. He is a process systems engineer by education with a Diplom in Chemical Engineering from the RWTH-Aachen University and PhD in Biochemical Engineering from the University of Porto. Before joining GSK, Moritz worked as a Lecturer at the School of Chemical Engineering and Advanced Materials at Newcastle University, where his research focused on the development of novel hybrid modeling methods and their application to enable more efficient process operation/design.
Sune Klint Andersen, Principal Scientist at Janssen Pharmaceutical Companies of Johnson & Johnson
Presented by Sune Klint Andersen, Principal Scientist at Janssen Pharmaceutical Companies of Johnson & Johnson
Sune Klint Andersen is Principal Scientist for Spray Drying at the Janssen Pharmaceutical Companies of Johnson & Johnson. He has a Ph.D. in Chemical Engineering. He has worked with pharmaceutical spray drying for 17 years within process development for R&D and commercial scale, application of Quality-by-Design and PAT, particle engineering, drying kinetics, aseptic spray drying and advantages and disadvantages of spray vs freeze drying processes. He previously worked at Novo Nordisk for 7 years.
Kim Li, PhD, DABT, MPH, Amgen Inc and Dries Cardoen, Team Leader of Study Directors at Nelson Laboratories, LLC
Presented by Kim Li, PhD, DABT, MPH, Amgen Inc.
Followed by an industry perspective presented by Dries Cardoen, Team Leader of Study Directors - Inhalation/topical/transdermal products at Nelson Laboratories, LLC
Permitted Daily Exposure (PDE) has origin in ICH Q3C: Impurities – Guideline for Residual Solvents. This webinar will discuss the principles and methods of applying PDEs to extractactables and leachables (E&L) impurities in pharmaceutical products. We will review the regulatory advice on the DO’s and DON’Ts. Then we will review the current toxicology risk assessment practice on data gathering, literature synthesis and selection of the critical studies with robust toxicity endpoints. We will show how the toxicity endpoints are transformed into chemical-specific PDE values with modifying factors. We will highlight the importance of clinical relevance to further refine the PDE values. For a case study, we will share an extraction study of a polyolefin bag for use with a lyophilized product. We will examine the risk matrix per USP Assessment of drug product leachables associated with pharmaceutical packaging/delivery systems. The toxicology assessment of the extractables profile of the bag will illustrate the derivation of chemical-specific PDEs, as well as the refinement by taking clinical relevance into consideration.
Niki Wong, Principal Research Scientist at AbbVie Operations Singapore Pte Ltd
Full Title: New Technologies for Improving and Controlling Product Quality, Expression, Timelines and Yield in Upstream Process Development
Presented by Niki Wong, Principal Research Scientist at AbbVie Operations Singapore Pte Ltd
Increasing product demands at competitive pricing drive the need for accelerated product approval timelines, with reduced manufacturing risks and costs. New technologies which can debottleneck upstream process development or improve manufacturability success in early phase process development, translate into cell culture processes with high yield and desired product quality. An overview of different strategies and recent perspectives in upstream process development will be presented.
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Big Data Strategies for Cell Therapy ManufacturingScott R. Burger, MD, Principal of Advanced Cell & Gene Therapy, LLC and Heidi Hagen, Co-founder and CSO for Vineti Inc[[ webcastStartDate * 1000 | amDateFormat: 'MMM D YYYY h:mm a' ]]75 mins