Autoimmune - We are Live! (11)

Day Two Agenda

7:30 am Check-In & Coffee

8:20 am Chair’s Opening Remarks

Utilizing Novel Technology & Methods to Streamline Development

8:30 am Biomarker Quests in an Evolving Landscape of Potency Assays

  • Jorge Burns Associate Professor, University of Ferrara


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

9:00 am Roundtable Discussion: Multi-Omics Approach to Biomarker Discovery & Potency Assay Developmen


• 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

9:30 am Leveraging NanoBiT-Based Potency Assays for Cell Therapy

  • Brock Binkowski Senior Research Scientist, R&D Protein & Nucleic Acid, Promega Corporation


  • 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

10:00 am Morning Break & Networking

10:01 am
Developing Potency Assays that are Fit for a QC Environment

10:30 am Cell Based Potency Methods in Quality Control


Cell Based Potency Methods in Quality Control

• Establishing potency methods as part of a control strategy

• Understanding QC requirements for potency methods

• Implementing cell-based potency testing in a QC environment

11:00 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


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


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

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


• 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

  • Fan Yang Vice President, Cell Therapy, Aisar Biotechnology


• 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


• Understanding the regulatory basis for the necessity of potency assays

• Discussing phase appropriate expectations regarding potency assays for cell therapy 


• 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, Analytical Development, Cell & Gene Therapies, Johnson & Johnson
  • Fan Yang Vice President, Cell Therapy, Aisar Biotechnology
  • Darren Kamikura Scientific Director, Potency & Functional Characterization, Bristol Myers Squibb
  • Christopher Storbeck Senior Scientific Evaluator, Health Canada


• 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