7:15 am Check In, Coffee & Networking

8:15 am Chair’s Opening Remarks

  • Tam Soden Executive Director & Global Head of Global Quality Control, Kite, a Gilead Company

8:20 am Panel Discussion: Discuss the Need to Standardize Across Industry to Benchmark Accurate Results

  • Nathan Majewski Associate Director, Cell Therapy Analytical Development, BMS
  • Sumona Sarkar Biomedical Engineer, National Institute of Standards & Technology
  • Richard McFarland President, Standards Coordinating Body Regenerative Medicine

Synopsis

• Discuss how to standardize common tests across different companies, different CROs etc. to ensure accuracy and comparability

• Determine an industry standardized control to evaluate the basic performance across different kinds of assays

Keynote Case Studies – Regulatory Experience to Hit Guidelines and Reduce Delays

8:50 am Validation of Flow Cytometry Methods in Cellular Therapies

  • Petra Pham Principal Scientist (Flow Cytometry), Global Product Quality, BMS

Synopsis

• Multi-parameter flow cytometry is a key analytical platform to measure identity, purity and strength of in-process drug substance and final drug product.

• Due to the complexity of the cellular drug product, lack of existing reference standards and appropriate calibration materials, limited regulatory guidelines, as well as the quasi-quantitative nature of the output, producing reliable and reproducible data is especially challenging in the context of a cGMP environment

• Here, we describe how the Breyanzi™ program addressed these challenges and contributed to a successful licensure. Validation runs were executed based on customized experimental design, and statistical analyses were performed for individual validation parameters to address requirements and concerns by Regulatory Authorities

9:20 am Industry Consideration for Hitting Regulatory Guidelines

  • Jas Uppal Chief Regulatory & Quality Officer, Gamida Cell

Synopsis

• Experience in approach to submitting filings with the FDA or other regulatory authorities

• Define the strategy and approach to get seamless acceptance of analytical protocols

• Share experience of common questions from regulatory boards regarding assays and how they were addressed

9:50 am Quality Control Flow Cytometry In-Process and Final Product Testing for Cell Therapies

Synopsis

• Review of key requirements to set up a commercial QC flow cytometry laboratory
including laboratory design and equipment
• Discussion of critical parameters for in-process and final product testing via flow
cytometry
• How to meet industry data integrity requirements on flow cytometry systems

10:20 am Speed Networking & Refreshment Break

Synopsis

As the analytical community is reunited, this valuable session will ensure you can reconnect with your peers in the room to make new and lasting connections. Running concurrently as an in-person and digital session, all attendees will have the opportunity to meet and network with their academic and industry colleagues.

11:00 am Developing a Successful Potency Assay for Complex Biologics

  • Knut Niss Chief Technology Officer, Mustang Bio

Synopsis

• Potency testing is critical to define how effective the product is, consistency of batches and safety

• Share views of regulatory authorities on the potency assay given the recent delays in submissions

• Experience in developing a robust assay to overcome variability when there is a lack of understanding of key attributes or mechanism of action

• Understand what is key to measure, how does analytics intersect with drug development?

11:30 am Advancing CAR-T Characterization with Droplet Digital PCR

Synopsis

• Droplet Digital PCR is being widely adopted for vector copy number discrimination
in CAR-T development and manufacturing
• Its high precision is ideally suited for molecular characterization
• Learn about its utility in vector copy number screening, gene-editing confirmation
and impurities detection

12:00 pm Regulatory Requirements and Best Practices for Cell Therapy Analytics

  • Michael Lehmicke Sr. Director, Science & Industry Affairs, Alliance for Regenerative Medicine

Synopsis

• Describe how regulatory expectations evolve from early to late stage development

• Provide an update on regulatory guidance

• A-Cell Preview – best practices for analytical assay development

• Potency assays – the Achilles heel of cell therapies

12:30 pm Utilization of Digital PCR for Quantification of CAR T Cell Products

  • Lindsey Murphy MD, University of Colorado and Children’s Hospital Colorado

Synopsis

• Digital PCR (dPCR) has many advantages compared to qPCR for quantification
of CAR T cell products including simplicity, reproducibility, lower limit of detection,
and absolute quantification
• Describe the development of a dPCR assay to specifically detect and quantify the
novel bicistronic CD19xCD22 CAR T cell product
• Demonstrate that digital PCR strategies can be utilized for accurate, precise, and
reproducible quantification of unique CAR transgenes and Vector Copy Number
(VCN) measurements

1:00 pm Lunchtime Refreshments & Networking

Early-Stage Cell Characterization

Next Generation Analytical Testing for Early- Stage Characterization

2:00 pm Leveraging the Power of Multimodal Analytical Platforms to Inform on Design and Characterization of Cell Therapy Products

Synopsis

• Engineering cellular therapies to address the unique challenges of each cancer requires the understanding of the challenges imposed by the tumor and the responses elicited when immune cells encounter tumor cells.

• Discuss the next-generation tools available for deepening our understanding of these interactions

• Describe potential strategies for implementing multimodal approaches to product characterization

2:30 pm Phase-Appropriate Development of Potency Assays for Starting Materials and UCART Cellular Therapy

Synopsis

• Potency assay development for UCART drug products is complex and often multimodal
• Designing phase appropriate potency assays to support product characterization and development
• Developing potency assays for starting material during early phase

3:00 pm Single-Cell Multi-omics to Accelerate Cell and Gene Therapy Characterization

  • Nori Ueno Associate Director, Cell and Gene Therapy Business Development, Mission Bio

Critical Quality Control

Benchmark Best in Class CQA Strategies

2:00 pm Lessons Learned From Kymriah QC Specifications

Synopsis

• Correlative analysis to determine the critical quality attributes of products

• Outline the CQA strategy, detailing what to control and what to measure

• Understand what makes an analytical assay QC friendly

2:30 pm Strategy and Analytical Tools to Ensure Genetic Identity & Integrity from Vector to CAR-T Product Development

Synopsis

– Building on a fully characterized stable cell line for viral vector production
– Building genetic identity & integrity packages using target locus amplification (TLA) and NGS
– Challenges and perspectives on genetic integrity testing for CAR-T manufacturing

3:00 pm Ensuring Safety to Achieve Rapid Release

Synopsis

• Discuss new tools that are required to fully characterize cell and gene therapy product purity, stability, and viability quickly, accurately, and simultaneously across all stages of biologic development
• Demonstrate how to use Aura for high throughput, low volume particle imaging to rapidly size, count, and identify biological particles
• Via membrane microscopy, effectively measure CQAs USP 788, 1046 and 1047 related to purity and stability, viability, and particulate controls
• Present case studies focused in subvisible particle content of AAV formulations at low volumes under different stress conditions, and a thorough particle characterization of cellular therapy products including cell aggregation, viability, and product impurities

3:30 pm Refreshment Break

Characterization Advances to Analyse Purity

4:00 pm Track Closed

4:30 pm Monitoring Product and Process Related Impurities

  • Pete Cotter Associate Director, Analytical Development, Vor Biopharma

Synopsis

• Describe the use of automated flow cytometer platform for cell-based impurities

• Share methods to monitor process related impurities in drug product

• Maximize the functionality of existing instrumentation

5:00 pm Cellular Impurities Quantification: How to Move Away from Flow Cytometry Analysis

Synopsis

– Evaluation of alternative approach to detect cellular impurities in the drug product
– How to increase sensitivity without losing in precision
– Perspective: implementation of DNA methylation based qPCR as QC routine test

 

Validating New Tools Fit for QC

4:00 pm Integrating QC Tools into Process Workflows

Synopsis

“Integrating QC Tools into Process Workflows instantly captures data without additional effort to expedite deviation handling and drive better analytics.”

Process Management software helps streamline manufacturing, improve materials management, and reduce errors.  Quality Management Systems help to improve quality, increase efficiency and minimize risk of non-compliance. When the two are integrated a myriad of additional benefits can be achieved including:

  • SOP, Job Aids & Forms Management
  • Deviation Management
  • Analytics & Reporting
  • Regulatory Compliance and Accreditation

4:30 pm Develop a QC SOP for New Tools

  • Tam Soden Executive Director & Global Head of Global Quality Control, Kite, a Gilead Company

Synopsis

• Discuss how to use RNA seq, high throughput data and real time PCR

• Describe how to write the QC SOP to set a threshold for new data analysis

• Understand the value of the data to determine how to utilize the technology as a way to capture CQA in a reliable fashion

5:00 pm Novel Tools and Benchmarking Techniques for Mammalian Cell Measurements: Enabling Inter-laboratory Comparability

  • Laura Pierce Biomedical Engineer National, Institute of Standards & Technology

Synopsis

• Discuss current efforts to apply higher levels of quantification to fluorescent cell images using control materials and how these tools will enable better comparability for international metrology inter-lab studies

• Describe our newly updated Cell Analysis Tool (COMET) and its application in the cell therapy field

• Summarize ongoing metrology efforts and new measurement tools in development

5:30 pm Chair’s Closing Remarks

5:35 pm Close of Conference