8:00 am Morning Refreshments & Networking

8:25 am Chair’s Opening Remarks

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

8:30 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

9:00 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

9:30 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

10:00 am Expertise Partner Presentation to be Confirmed

10:30 am Structured 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.

11:30 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?

12:00 pm Expertise Partner Presentation from Bio-rad

12:30 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

1:00 pm Expertise Partner Presentation from QIAGEN

1:30 pm Lunchtime Refreshments & Networking

Early-Stage Cell Characterization

Next Generation Analytical Testing for Early- Stage Characterization

2:15 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

2:45 pm Development of Potency Assays for Early-Phase Lentivirus-Based Gene Therapy

Synopsis

• The potency assays for lentiviral vector lots are designed based on manufacture of gene therapy products

• Multiple potency assays are developed in the early stage of product life cycle

• The potency assays for gene therapy products may provide insights into analytical testing for late-phase lentiviral vector

3:15 pm Program Partner Presentation

Critical Quality Control

Benchmark Best in Class CQA Strategies

2:15 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

2:45 pm Ensuring Safety to Achieve Rapid Release

  • Sarah Snykers Director, Cell Therapy Manufacturing Unit, Celyad

Synopsis

• Release for inclusion vs final disposition, determine the risk benefit of safety testing and timings

• Extensive biomarker analysis

• Discuss how to release autologous products as fast as possible for patients

3:15 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

3:45 pm Refreshment Break & Poster Session

Characterization Advances to Analyse Purity

4:30 pm Advances in Phenotyping to Analyse Purity, Identification & Quantification

  • Sarah Snykers Director, Cell Therapy Manufacturing Unit, Celyad

Synopsis

• Deep, granular T cell phenotyping to explore multiple markers

• Characterization assays to collate information about the drug product phenotype throughout the life cycle of drug development

• Assess product specific characterization

• Quantifying core panels of immunomodulatory cytokines and inflammatory cytokines to define the product phenotype

5:00 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

Validating New Tools Fit for QC

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