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
Synopsis
- 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
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
9:30 am Leveraging NanoBiT-Based Potency Assays for Cell Therapy
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
10:00 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
Synopsis
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
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
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
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
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
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?