Pre Conference Workshop Day

Monday December, 5 | Boston, MA

Workshop A

10:00 am Flow Cytometry 101: Exploring the Design & Practical Considerations for Best-in-Class Flow Cytometric Analysis

  • Petra Pham Associate Director, Flow Cytometry & Global Product Quality, Bristol Myers Squibb
  • Sean Bradley Principal Scientist, Analytical Development, Catamaran Bio

Synopsis

Flow cytometry is a powerful technique commonly used to detect and evaluate many characteristics of cell populations. As it is arguably the most important technique during multiple stages of analytical development and quality control, one key to success is truly understanding all aspects of set-up, design and validation that a GMP-compliant process entails. Attend to:
  • Learn to set-up, develop, qualify and validate a flow cytometry assay for product characterization, release and stability
  • Discover ways to use flow cytometry for surrogate functional assays to allow comparison and release of cellular products
  • Explore basic gating principles alongside more complex assay characteristics to learn as much as possible about your cells of interest

Workshop B

10:00 am Streamlining & Optimizing Viral Vector Bioprocess Analytical Development for Engineered Cell Therapy Products

  • James Richardson Senior Director, Analytical Development, Interius BioTherapeutics
  • Ho-Tak Lau Senior Scientist, Process Analytics, Umoja Biopharma

Synopsis

Broadly speaking, analytical development for cell therapies can be divided into drug product analytics and gene delivery analytics. With the former being the main focus of the industry, it is important to not forget the importance of the latter when engineered cell therapy products are a common occurrence. This workshop is a one-stop-shop to understand the main considerations and to simplify your analytical development and quality control strategies for viral vectors.

Attend to:

  • Review the common analytical tests and regulatory expectations for viral vectors used to engineer cellular therapeutics
  • Learn the basics in assay development, qualification and validation for gene delivery analytics
  • Discuss the strategy and analytical tools used to confidently characterize the vectors and ensure genetic integrity of the final cell therapy product

Lunch Break & Networking

Workshop C

1:00 pm Investigating Process & Product Related Impurity Testing of Cellular Therapeutics

  • Anthony Leyme Principal Scientist, Analytical Development, Elevate Bio
  • Taylor Zhang Executive Director, Product Characterization & Analytical Chemistry, Sana Biotechnology

Synopsis

In the course of manufacturing, chemicals may be added upstream as part of the bioprocess to improve product expression and recovery; of course, these are removed but some residuals remain. Equally, there may be product related impurities for example, host cell protein, host cell DNA, N-terminal truncations etc. Therefore, assays must be employed to ensure that any impurities present are below pre-determined levels.

Attend to:

  • Understand best practices to reduce or eliminate impurities to acceptable levels for risk mitigation
  • Learn to identify and quantify impurities for process consistency and product purity
  • Outline tools used to measure and characterize impurities, including mass spectrometry – how can we develop more rapid testing methods?

Workshop D

1:00 pm Developing Successful Potency Assays to Determine Activity of Your Cell Therapy Product

Synopsis

There is no single test that can adequately measure the product attributes that predict clinical efficacy; therefore, developing a successful potency assay is notoriously hard to accomplish. Additionally, to successfully develop a potency assay, many challenges must be overcome, including the inherent variability of starting materials, limited lot size and material for testing, limited stability, lack of appropriate reference standards, complex mechanism of action(s) and more.

Attend to:

  • Learn to develop a potency assay matrix that involves evaluating two or more mechanisms of action
  • Address potency not only for CAR-T, but for gamma delta T cells and other cell therapy drug products that possess different mechanisms of actions
  • Understand how to set up controls and a reference standard, without a golden standard
  • Prepare for BLA submission by exploring the best ways to validate both quantitative and qualitative potency assays

Refreshment Break & Networking

Workshop E

3:30 pm Outlining Key Regulatory Considerations for Industry to Avoid Regulatory Pushback

  • Mallory Scott Associate Director, Regulatory CMC, Legend Biotech
  • Victor Lu Senior Vice President, Head of Regulatory Affairs, Innovative Cellular Therapeutics

Synopsis

The cell therapy field offers remarkable opportunities for overcoming the limitations of other therapies. However, this field is new and constantly evolving, accordingly there are many unique biological, clinical and regulatory challenges. This workshop focuses on the latter and will open the channel of conversation between regulatory experts and those involved in cell therapy research.

Attend to:

  • Define the best approach to submitting filings with regulatory bodies (i.e. FDA & EMA) to get seamless acceptance of analytical protocols
  • Address the need for consensus on core regulatory elements for the sound development of cell therapy products
  • Share experience and advice regarding key pain points

Workshop F

3:30 pm Utilizing Phase-Appropriate Automation to Reduce Costs Later in Development

  • Ajit Kamath Senior Scientist I, Analytical Development, Beam Therapeutics
  • Minnal Gupta Sequencing & Assay Automation Lead, Center for Cellular Immunotherapies, University of Pennsylvania

Synopsis

A major bottleneck that needs to be addressed for the successful commercialization of cellular therapeutics are the scale-up of manufacturing processes and analytical methods, and for autologous, the management of apheresis-related activities. To do this successfully and deliver more robust and effective therapies to patients, automated technologies need to be utilized from the early stages of development.

Attend to:

  • Discuss the need for phase appropriate automation to reduce assay variability and increase robustness
  • Consider step wise automation of assays to improve variability and reduce costs incrementally
  • Optimize the process for critical analytical tests – at what points should we implement automation to achieve this?
  • Learn about more recent advances in automated analytical tools, discussing potential regulatory implications