Round Table Discussions

Join a breakout discussion group. These are informal, moderated discussions with brainstorming and interactive problem solving, allowing participants from diverse backgrounds to exchange ideas and experiences and develop future collaborations around a focused topic.

TUESDAY, AUGUST 13 | 4:45-5:45PM

Optimizing Cell Culture Technology

Commonwealth Hall

TABLE 1: Viral Vector Production - The Grand Challenge of Gene Therapy

Moderator: Lorenz Mayr, PhD, CTO, Life Sciences, GE Healthcare

• Optimisation & scale-up of upstream packaging processes
• Optimisation & scale-up of downstream purification processes
• Miniaturisation & automation of viral vector production
• In-process control and digital tracking of production steps
• QC and product release criteria for viral vectors

TABLE 2: Glycan Composition Variation during CHO Production Processes

Moderator: Xiaotian Zhong, PhD, Senior Principal Scientist and Lab Head, Pfizer BioTherapeutic Research

• Control of the glycosylation profiles is essential for biopharmaceutical production
• With rising interest in producing therapeutic proteins with distinct N-glycan structures, these issues seem critical
• manipulation of culture conditions (temperature, pH, and feeds)
• glycol-engineering (i.e. generation of knockouts, and expression of glycan remodeling enzymes)

TABLE 3: Process Intensification Strategies

Moderator: Stefan R. Schmidt, PhD, MBA, COO and Head of Operations, BioAtrium AG

• Compressing process run times
• Continuous processing
• Combining unit operations
• Outsourcing of preparation efforts

TABLE 4:  Monitoring Continuous Culture Processes

Moderator: Michael Butler, PhD, CSO, Cell Technology, National Institute for Bioprocessing Research and Training (NIBRT)

• Are there alternatives to cell sampling?
• Are capacitance measurements a substitute for trypan blue?
• Are automated visual methods sufficiently well developed?
• Are there advantages in detecting an early onset of apoptosis?

TABLE 5:  Omics and Big Data

Moderator: Jamey Young, PhD, Associate Professor, Chemical and Biomolecular Engineering, Vanderbilt University, and Co-Founder and Chief Scientific Officer, Metalytics LLC

• Which technologies have been most useful for specific applications?
  o Process development
  o Media development
  o Cell line development
  o Biosimilars development
  o Regulatory compliance
• What are your major success stories with use of omics and big data?
• What are the important limitations and challenges to consider?
• What are the emerging trends and latest developments in this space?
  
  

Continuous Processing in Biopharm Manufacturing

Commonwealth Hall

TABLE 6: Comparison and Selection Guidance for MCC System in Continuous Downstream Processing

Moderator: Louise Taylor, BSc., Downstream Scientist, Biologics, Centre for Process Innovation

• The differences: max columns, max flow rate, price, software, hardware, brand name
• The applications: clinic/commercial, quantity, stability, upstream process
• The one: why, when, how
• The future: the ideal MCC system

TABLE 7: What You Need to Know About End-to-End Continuous Bioprocessing

Moderator: Robert Dream, Managing Director, HDR Company LLC

• Challenges and opportunities
• Need-based analysis
• Continuous biomanufacturing implementation now and in the future
• Is continuous manufacturing the best solution for your biotech facility?
• Continuous manufacturing as a tool for process intensification

Host Cell Proteins

Commonwealth Hall

TABLE 8: HCP for Gene Therapies

Moderator: Emily Menesale, Senior Associate Scientist, Analytical Development, Biogen

• What are some logistical challenges in running HCP assays for gene therapy products as opposed to other types of drugs?
• What are some challenges in using non-traditional host cell lines? What cell lines are people using and what are their strategies?
• Are commercial HCP kits ok for non-traditional host cell lines or are custom HCP assays needed?
• Have people observed any problematic viral antigens?

TABLE 9: Best Practices in HCP Immunoassay Development and Qualification

Moderators: Eric Bishop, Vice President, R&D, Cygnus Technologies

• Common misconceptions and mistakes in developing HCP assays
• What Orthogonal Methods to use to demonstrate an HCP assay fit for purpose?
• Generic vs Process-specific HCP Assays

Rapid Methods to Assess Quality & Stability of Biologics

Commonwealth Hall

TABLE 10: Novel CQAs for Novel Formats

Moderator: Elisabeth Kast, PhD, Senior Scientist, Protein Analytics, AbbVie

• When in the discovery/development timeline do you identify non-traditional CQAs for a fusion protein or novel modality?
• How do you rapidly implement monitoring during development if no platform method is available?
• Does MAM play a role in your strategy for novel formats?

TABLE 11: Use of Risk-Based Predictive Stability Tools in Regulatory Filings

Moderator: Kara Huang, PhD, Senior Scientist, Drug Product Development, AbbVie

• Types of tools available
• Potential pitfalls in using these tools
• Have any of these tools been successfully used in a regulatory filing to justify reduced testing?

TABLE 12: Protein Aggregation in Novel Biologics

Moderator: Subhashchandra Naik, PhD, Senior Scientist, Reform Biologics

• Importance of mechanistic understanding of protein degradation and aggregation pathways
• Predictive tools and characterization methods
• Impact of aggregation on product development timelines

Cell Therapy CMC and Analytics

Commonwealth Hall

TABLE 13: Accelerating Cell Therapy Development

Moderators: Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

• Most common questions asked by companies/ regulators
  
• Lessons learnt from recently approved products
• Challenges around gene-edited cell therapies
• Preparing for IND – What are the priorities, preclinical packages?
• CAR Ts in combination with other agents – CMC implications

TABLE 14: Plasmid Raw Material Control Strategy from Discovery to Commercial

Moderator: Lawrence Thompson, PhD, Principal Scientist, Analytical Research & Development, BioTherapeutics Pharm Sciences, Pfizer

• In Sourcing vs Out Sourcing
• Release Testing & Specifications
• Method Lifetime: Development, Qualification & Validation
• Stability & Period of Use
• Storage Temperature & Container Closure
• Critical Material Attributes & Comparability

TABLE 15: Allogeneic vs Autologous

Moderator: Michael D. Jacobson, PhD, Managing Partner, Cambridge Biostrategy Associates

• How to manage scientific, technical and clinical risks?
• Manufacturers: choosing between allogeneic and autologous; or can you do it all?
• Suppliers: how to invest in an uncertain future?
• What is the roadmap for combining autologous and allogeneic approaches? Will allogeneic eventually replace, or complement autologous?
• New technologies (iPS, NK cell, etc.) - how will these impact current CAR-T and TCR approaches?

Process Characterization and Control

Commonwealth Hall

TABLE 16: Could Artificial Intelligence and Machine Learning Accelerate Drug Development? How and What is Needed?

Moderator: Wolfgang Paul, PhD, Principal Scientist and Digitalization Lead, Large Molecule Research, Roche, Germany

• AI in Drug design and development - what does this mean? Hype or reality?
• Prediction in protein design & development - expectations
• Is acceleration possible or is AI more an enabler?
• Critical prerequisites to enable machine learning and prediction in drug design and development
• Benefits measurable and  which KPI's have to be addressed?

FRIDAY, AUGUST 16 | 7:30-8:20AM

Optimizing Cell Line Development

Harborview 1

TABLE 1: Industry/Academic Collaboration

Moderator: Hooman Hefzi, PhD, Postdoctoral Researcher, Pediatrics, UC San Diego (UCSD)

• What makes a good collaboration?
• What are the largest barriers to collaboration?
• Are there alternatives to traditional collaborative setups?

TABLE 2: What a Stability Study Package Should Consist of for STABLE Producer Cell Lines

Moderator: Aubrey R. Tiernan, PhD, Senior Scientist I and Head, Cell Line Development, Ultragenyx Gene Therapy

• What affects stability?
• What does a stability study package look like?
• How can we use genome stability information to generate more stable cell lines?
• What are some strategies to evaluate long term stability?
• What new technologies can we use to predict clone stability?

TABLE 3: Cancelled

Detection, Characterization and Control of Impurities in Biologics

Harborview 3

TABLE 4: Challenges in Antibody Production

Moderator: Michael Anyadiegwu, PhD, Senior Scientist, Downstream Processing, Centre for Process Innovation Ltd., National Biologics Manufacturing Centre

• Streamlining processes for the purification on antibodies and new medicines
• New tools and methods

TABLE 5: Accelerated Development and Risk Management for Unmet Medical Needs and Breakthrough Therapies

Moderator: Douglas E. Kiehl, MSc, Research Advisor, Bioproduct, Research & Development, Eli Lily and Company

• Accelerated development of pharmaceuticals for unanticipated/unmet medical needs
• Accelerated regulatory review/approval processes for unmet medical needs and breakthrough therapies
• New technologies for detection of pathogens and impurities, also in-vitro and companion diagnostics

Process Characterization and Control

Harborview 2

TABLE 6: Analytical Challenges in Cell Therapy

Moderator: Sheila G. Magil, PhD, Managing Director, Industry Specialized Services, BDO

• Using OOS materials for patients
• What release assays are needed for cell therapy products?
• Quality and testing of raw materials used for manufacture of cell therapy
• What does purity mean for a cell therapy product?

TABLE 7: Challenges and Opportunities during Implementation of New Analytical Technologies

Moderator: Elena Smith, PhD, Deputy Director, Quality Control, Sanofi Pasteur

• Alternative standards
• Alternative methods
• Challenges for the analytical characterization for the new technologies

TABLE 8: Analytical Comparability for Innovator Products - How Much is Enough?

Moderator: Sonia Taktak, PhD, Principal Scientist, BioTherapeutics Pharmaceutical Sciences, Pfizer, Inc.

• How should one design a comparability study in early/late stage development vs. commercial?
• What stability/stress conditions do you include in your comparability studies?
• Should one use statistics for assessing comparability/evaluating rates or routes of degradation?
• What analytical characterization methods do you include in your comparability studies?

Gene Therapy Manufacturing

Harborview 2

TABLE 9: Scale-Up of Gene Therapy Products

Moderator: Nathalie Clément, PhD, Associate Director and Associate Professor, Powell Gene Therapy Center, Pediatrics, University of Florida

TABLE 10: Purification of Viral Vectors

Moderator: Alois Jungbauer, PhD, Professor, Institute of Biotechnology, University of Natural Resources and Life Sciences (BOKU), Matthew Roach, PhD, Process Development Engineer, Precision Biosciences