Day Two Agenda

8:00 am Check-In & Coffee

8:50 am Chair’s Opening Remarks

Utilizing Novel Technology & Methods to Streamline Development

9:00 am Biomarker Quests in an Evolving Landscape of Potency Assays

  • Jorge Burns Associate Professor, University of Ferrara

Synopsis

  • Identifying osteogenic biomarkers, a case study brings surprises
  • Improving mimicry of in vivo metabolism
  • Developing surrogate potency assays with 3D spheroid models

9:30 am Leveraging Transcriptional Genomics for Identification of Potency Markers for Allogeneic Cell Therapies

Synopsis

• Developing strategies for biomarker discovery using NGS platforms

• Validation of markers using various protein screening methods 

• Identifying selection criteria for final panel of marker(s) based on RNA/protein data 

10:00 am Leveraging NanoBiT-Based Potency Assays for Cell Therapy

  • Brock Binkowski Senior Research Scientist, Research and Development Protein and Nucleic Acid, Promega Corporation

Synopsis

  • Measure target cell specific killing using HiBiT Target Cell Killing assays
  • Cell lines with target knockouts as system suitability controls
  • Sensitive, homogeneous assays for assessing cytokine secretion using Lumit® Immunoassays

10:30 am Morning Break & Networking

CAR-T & TCR-T

Developing Potency Assays that are Fit for a QC Environment

11:00 am Cryopreserved Ready-to-Use Target Cells to Improve Control of QC Potency Methods for Autologous CAR-T Therapies

  • Darren Kamikura Scientific Director, Potency & Functional Characterization, Bristol Myers Squibb

Synopsis

• Improving potency method precision and control through 

single-use aliquots of target cells

• Developing robust conditions for expansion and 

cryopreservation of target cells

• Qualifying new lots of banked material for use in routine 

testing 

11:30 am Challenges for Potency Assays with a Platform Approach Across Allogeneic CAR-T Cell Products

  • Xenia Peluffo Naj Associate Director & Principal Scientist - Analytical Development & Program Manager, Cellectis Stem Cells

Synopsis

• Developing a platform approach for potency assays across 

different products

• Choosing elidgible analysis model for potency assays 

• Setting system suitability criteria considering expected level of 

variability for allogeneic product

Other Cell Types

  • John Yu Director, Sana Biotechnology

Developing Potency Assays that are Fit for a QC Environment

11:00 am Cell Based Potency Methods in Quality Control

Synopsis

• Establishing potency methods as part of a control strategy 

• Understanding QC requirements for potency methods

• Implementing cell-based potency testing in a QC environment 

11:30 am A DOE Approach to Potency Assay Development for an Allogeneic NK Cell Product

Synopsis

• Reviewing the difficulties of potency assay development for 

allogeneic cell therapies which have high lot-to-lot variability 

• Discussing the impact of donor and assay variability on 

potency method optimization 

• Developing assays that are fit for a QC environment

12:00 pm Lunch Break & Networking

Carrying Out an Effective Validation Plan to Ensure Robustness

1:00 pm Adopting a Phase-Appropriate, Time & Cost-Effective Strategy for Method Validation for Potency Assays

  • Shibani Mitra-Kaushik Head, Product Control Analytics, Cell Therapies Cell Therapies Analytical Development, Takeda Pharmaceutical Co. Ltd.

Synopsis

• Identifying the best analytical methods to define potency assays reflective of the 

mechanism of action 

• Implementing validated potency assays for release, stability, and comparability

• Bridging challenges between early and late phase potency indicating methods

1:30 pm Validation Strategy of Potency Assay for a Phase III MSC Product

Synopsis

• Discussing the major challenges of potency assay for stem cell therapy

• Developing a quantitative, relevant, fast potency assay

• Outlining strategies to redefine and validate the potency assay

2:00 pm Afternoon Break & Networking

Developing a Potency Assay to Obtain Regulatory Approval

2:30 pm Potency Assays for Cell Therapy Products: A Regulatory Perspective

Synopsis

• Understanding the regulatory basis for the necessity of potency assays

• Discussing phase appropriate expectations regarding potency assays for cell therapy 

products

• Identifying challenges for potency assays for cell therapy products

• Optimizing potency assay design for cell therapy products

3:00 pm Panel Discussion: Late-Stage Potency Assay Development & Preparing for BLA

  • Ramon Mendoza Scientific Director chemistry, manufacturing & controlsTechnical Strategy for Advanced Cell & Gene Therapies, Johnson & Johnson
  • Fan Yang VP, Aisar Biotechnology
  • Darren Kamikura Scientific Director, Potency & Functional Characterization, Bristol Myers Squibb
  • Christopher Storbeck Senior Scientific Evaluator, Health Canada

Synopsis

• What are the important considerations for developing a potency assay fit for a commercial product? 

• What learnings can we take from products that are already commercialized? 

• What are the regulators looking for when submitting BLA?

3:45 pm Chair’s Closing Remarks

4:00 pm End of Conference Day 2