Breakout Discussions

Monday, 4:45-5:30

Optimizing Cell Culture Technology
Constitution A

Collecting, Managing and Analyzing Cell Culture Data

Moderator: Neil A. McCracken, P.E., MS, Associate Senior Consultant Engineer, Bioproduct Research and Development, Eli Lilly and Company

Common in-line and at-line analytical methods
Best practices for collecting and managing data from various in-line and at-line instruments
Best practices for analysis of data through tools like traditional fitting or more complex PLS/PCA

CQA Risk Assessment for Attributes Associated with Potency

Moderator: Taro Fujimori, PhD, MBA, Associate Director, Protein Analytics, Science and Technology Biologics, Abbvie Bioresearch Center

How does your company define the primary mechanism of action for a biotherapeutic? Who in your company is involved in these discussions?
What data are generated to support the primary mechanism of action? When are these data collected?
How does your company handle attributes that impact biological activities but are not associated with the primary mechanism of action? For example, signal inhibition and ADCC activities for an antibody drug conjugate.
How are bioassays used to assess critical quality attributes?

What are the Challenges your Organization is Facing in Deploying a QBD Approach for Development and Commercialization of Biopharmaceuticals?

Moderator: Naveen Pathak, PhD, Director, Process Development, Shire

Challenges in demonstrating the QbD value proposition
The challenges of deploying QbD approach for Accelerated and Breakthrough Status programs
Managing differences in QbD approach for development versus legacy products in your organization
Having the right infrastructure for QbD deployment and connectivity to the quality system

The Pros and Cons of using Transient Material vs Material Generated for a Stable Cell Line

Moderator: Joan Hilly Foster, MS, Senior Field Application Scientist, MaxCyte, Inc.

Proof of Concept studies, Assay Development, Purification Procedures, Safety Studies and Clinic Trials
Each step on the journey to the clinic will be discussed whether transient material is a feasible route
Does it help to mitigate risk and money for a company when a molecule fails in the clinic?
Is transient transfection a quicker way to the clinic without the time spent to generate a stable cell line?
Will the FDA even approve a transiently produced product?

Accelerating Biologics Development: Can Transient Expression Systems Speed Candidate Development?

Moderators: Henry C. Chiou, PhD, Director, Product Development, Thermo Fisher Scientific

Jonathan Zmuda, Director, Cell Biology, Thermo Fisher Scientific

Can transient expression systems be predictive of the characteristics of proteins expressed in stable cell systems?
What are some factors that can help align transient expression systems to downstream production systems?
When is the optimal time to transition from transient to stable expression?

Continuous Processing in Biopharm Manufacturing
Back Bay A

Regulations & Continuous Manufacturing

Moderator: Robert Dream, Managing Director, HDR Company

No specific regulations or guidance for continuous manufacturing, other than the definition of “lot”
Continuous manufacturing consistent with FDA’s QbD efforts
Potential to improve assurance of quality and consistency of drugs
Enables quality to be directly built into the process design

Bioprocess Economics, Understanding the Impact of Process and Technology Choices

Moderator: Andrew Sinclair, President & Founder, Biopharm Services Ltd.

Impact of single use technology in commercial greenfield manufacturing
What is single use role within a legacy stainless steel environment
What role does perfusion play compared to fed batch
Where how does continuous processing add value
Deconstructing the cost of cell therapies

Challenge and Potential Solutions for Continuous Processing

Moderator: Ji Zheng, PhD, Associate Director, Biologics Development and Manufacturing, Celgene

Current status of Industry on continuous processes and next generation continuous processing
Drivers of the continuous process
Intensified vs continuous (pros/cons)
Challenges for continuous downstream processing and virus clearance study
Challenges in scale up / implementation of continuous manufacturing
Control sampling plan for continuous processing
Perfusion can improve molecules quality attributes and How to link long perfusion processes to the development of a DSP
PAT role for continuous process

Host Cell Proteins
Back Bay B

HCP Regulations and Standards

Moderators: Maura Kibbey, PhD, Director, Global Biologics, U.S. Pharmacopeia

Erika Friedl, PhD, Quality Expert, Hematology and Transfusion Medicine, Paul-Ehrlich-Institute

HCP regulatory expectations throughout development
Common pitfalls
Current standards and upcoming developments
European vs. US expectations

Best Practices for HCP Analysis using Mass Spectrometry

Moderators: Christopher Yu, PhD, Principal Scientist, Genentech, A Member of the Roche Group

Veronika Reisinger, PhD, Lab Head, Biologics Technical Development and Manufacturing, Novartis

Best Practice for Quantitation
Choice of Instrumentation
LC/MS for direct lot release
Digestion Methods Optimization
Problematic HCPs, Next Gen HCP Analysis

Rapid Methods to Assess Quality & Stability of Biologics
Back Bay D

Impact of Statistics on Subvisible Particle Quantification

Moderator: Marina Gühlke, PhD, Analytical Scientist, Coriolis Pharma

How statistically relevant are particle concentrations obtained from low volume, microliter-sized samples?
Do particle number limits of USP<787>/<788>/<789> include measurement uncertainties?
Does pooling of low volume drug products solve our statistical issue or does pooling introduce more uncertainty?

How Do We Address Analytical Challenges With Novel Formats And Drug Delivery Platforms?

Moderator: Scott H. Chamberlain, Senior Research Associate, Protein Analytical Chemistry, Genentech

What are the early development challenges encountered?
How do we develop rapid platform methods?
How do we reconcile results from multiple assays of novel formats which often use indirect methods of measuring attributes?

New Methods for Prediction of Protein Aggregation During Storage for Lyophilized Products Based on Initial Release Data

Moderator: Lauren Fontana, Graduate Student, Pharmaceutical Sciences, University of Connecticut

Measurement techniques: Solid state amide hydrogen/deuterium exchange with mass spectrometric analysis (ssHDX-MS), solid-state nuclear magnetic resonance spectroscopy (ssNMR), Neutron back scattering and others
Prediction of formulation stability rank order prior to "accelerated" or real-time assays: Accuracy, limitations
Pros & cons of various techniques: Ease of use, sample limitations, assay time, sample size

Overcoming Formulation Challenges for Biopharmaceuticals Development
Back Bay C

Product Differentiation Through Innovative Formulation

Moderator: Jan Jezek, PhD, CSO, Research & Development, Arecor, Ltd.

How to deal with an increasingly complex formulation patent landscape
Need for product differentiation in the increasingly competitive biologics market
Enabling successful products through innovative formulation

How to Incorporate Excipient Quality and Specification Limits in Formulation Robustness Studies

Moderator: Helen Sjögren, PhD, Principal Scientist, Global Pharmaceutical R&D, Ferring Pharmaceuticals

Surfactants, how to control quality, levels and stability in drug product – regulatory requirements
Strategies for in-use stability, focus on infusion products with preparation steps before administration

Screening Methods That Are Most Indicative of Final Formulation Parameter and Most Useful in Formulation Development and Selection

Moderator to be Announced

What screening method(s) are the most predictive of formulation stability?
Are there new methods/instruments that have the potential to be a “step change” in the way formulation screening is conducted?
How do you probe high concentration behavior with limited sample available for screening?

Understanding the Role and Effect of Buffers in Biopharmaceutical Formulation Development

Moderator: Shreya Kulkarni, PhD Candidate, Pharmaceutical Sciences, University of Connecticut

Importance of buffers in formulation design and stability.
Do buffer ions play a role other than buffering? (eg. by interacting with ionized residues on the protein)
Is it possible to completely strip ions during buffer exchange? (Donnan equilibrium)
What is the effect of buffers on drug product stability?

Cell Therapy CMC and Analytics
Constitution B

Accelerating Cell Therapy Development

Moderators: Fouad Atouf, PhD, Vice President, Global Biologics, USP

Matthias Renner, PhD, Scientist, Federal Institute for Vaccines and Biomedicines, Paul Ehrlich Institute

Bernadette Keane, PhD, Principal, Keane Consultancy

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?
“Reverse translation” to improve manufacturing and development outcomes
CAR Ts in combination with other agents – CMC implications

Commercial Manufacturing of Gene-Modified Cell Therapies: The Challenges Ahead

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

What are the most important problems that need to be solved over the next 3-5 years for this industry to thrive?
What key innovations would you like to see that you believe would be transformative?
How low do COGS need to go, and how (and when) will we get there?
How automated and integrated will cell therapy manufacturing become over the next 5-10 years? What parts of the workflow are most ripe for integration, and which are not?

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