Day Two Agenda

8:00am - 4:30pm | London, UK

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8.00 am Registration Open & Coffee Networking

8:50 am Chair’s Opening Remarks

Discovering New Technology to Expand & Improve the Analytical Toolbox

9:00 am Cell and Gene Therapy Products Characterisation Using Next-Generation Sequencing

Synopsis

  • Understanding the advantages and challenges of next-generation sequencing (NGS) based methods
  • Providing rich information about genetic variants, transcriptome dynamics and more • Exploring best practice to perform NGS based transcriptomic data analysis
  • Addressing the requirements for assay qualification

9:30 am Developing ddPCR Based Assays for CAR T Cell Product Release: An India Perspective

Synopsis

  • Accurate measurement of VCN is crucial for product release of genetically modified cellular therapies or CAR T cells intended for infusion
  • Utilizing droplet digital PCR for enumerating genetic modification events per genome
  • Evaluating the in vivo persistence of infused CAR T cells in short- and long-term follow up of the subjects enrolled in the clinical trial

10:00 am Implementation of the ELLA Platform for Routine Testing During Assay & Process Development of UCART Products

Synopsis

  • Sharing Cellectis’ experience of ELLA as a tool supporting process development
  • Replacing ELISA with ELLA assays: a case study
  • Exploring the pros and cons of ELLA technology

10:30 am Morning Break & Networking

Validating Analytical Methods to Fulfil GMP Guidelines

11:00 am Golden Lessons of Cell Therapy Method Development & Validation

  • Ruti Goldberg Associate Director, Bioassays and Translational Medicine, Pluristem Therapeutics

Synopsis

  • Evolving validation requirements and analytical methods through the product’s life cycle, from early-stage development to commercial use
  • Transferring analytical methods from developers to quality control groups to ensure proper validation is performed to justify their intended use
  • Reviewing case studies of analytical method development and validation in cell therapy through the product’s life cycle

11:30 am The Development & Validation of a Flow Cytometry-Based Method: Best-in-Class Approaches

Synopsis

  • Outlining approaches to establish full validation of a well-controlled flow cytometry assay
  • Reviewing the challenges in validation e.g. the inherent biological variability of cells, the limited stability of samples, the requirements for cytometer set-up and the complexity of the data output and interpretation of result
  • Verifying the performance characteristics of the assay are suitable, reliable, and reproducible

12:00 pm Panel Discussion: Validating Analytical Tools to Streamline Movement from Pre-Clinical (non-GMP) to Clinical (GMP) Development

  • Benoit Champluvier Executive Director, Process & Analytical Development, Cellectis
  • Ruti Goldberg Associate Director, Bioassays and Translational Medicine, Pluristem Therapeutics
  • Emilio Cosimo Product Development Manager, TC BioPharm

Synopsis

  • Discussing how to best validate different analytical methods in GMP: flow cytometry, next-generation sequencing, PCR and more
  • Managing tech transfers to manufacturing sites at various stages of development

12:30 pm Lunch Break & Networking

Share Ideas to Learn from Others to Streamline Analytical Development

1:30 pm Roundtable Session: Discussing Challenges Around Stability Requirements and Testing for Cell Therapies

Synopsis

  • Does the regular long term stability programs for frozen product provide meaningful information?
  • Does post-thaw degradation (e.g., room temperature stability over 24h) and analysis of both degradation rate and time to impact on the cells give more insights?
  • Do we need to change our habits on how to report the stability? The stability results need to be compared to T=0 but due to lack of statistical power the statistical analysis and p-values should be used with caution. Preferable to understand the impact of freezing/thawing stress on the biology of the cells and whether there is any impact on the cell population of interest.

2:00 pm Roundtable Session: Managing the Heterogeneity of Starting Materials to Produce Consistent Products

Synopsis

  • To what extent is cell heterogeneity an issue for cell-based ATMPs?
  • What methods can we use to measure and manage variability?
  • How do we ensure consistent production of high-quality therapeutics while donor-to-donor variability is present?

2:30 pm Afternoon Break & Networking

3:00 pm Employing Automation to Shorten the Process for Critical Analytical Tests

Synopsis

  •  Reviewing current tests for release which provide a bottleneck to timeline
  • Leveraging automation to circumvent these bottlenecks and reduce time to release and administration

3:30 pm Creating an Automation Roadmap for Cell Therapy Testing

  • Tam Soden Executive Director, Global Head of Quality Control, Kite: A Gilead Company

Synopsis

  • Starting with the end in mind: utilizing automation to speed up release and reduce costs in a commercial QC environment
  • Outlining more recent advances in automated analytical tools and utilization
  • Exploring the main challenges and considerations of replacing manual-based methods with automation, discussing potential regulatory implications

4:00 pm Chair’s Closing Remarks & End of Conference