Conference Day One
Wednesday, March 12, 2025
8:30 am Check-In, Coffee & Light Breakfast
9:25 am Chair’s Opening Remarks
Optimizing Potency Assay Development Strategies for Enhanced Assay Robustness & Validity
9:30 am Successes & Challenges in Designing a Potency Assay Matrix for Early Stage Parkinson’s Cell Therapy
Synopsis
- Optimizing assay validation strategies for products in later stage clinical trials
- Determining how clinical companies approach assay validation to regulatory approval
10:00 am Are you ready for GMP? How to optimize your potency assay for clinical and commercial success
Synopsis
- Comprehensive Potency Assurance: Best practices for developing, transferring, qualifying, and validating GMP potency assays across the product lifecycle.
- Overcoming Assay Challenges: Addressing variability in cell populations, defining potency attributes, and ensuring assay sensitivity and specificity.
- Regulatory & Real-World Insights: Case studies and strategies to align with the latest and continually evolving regulatory expectations.
10:10 am Morning Refreshments & Speed Networking
Synopsis
Connect with all your fellow analytical peers! This is your opportunity to network and forge new contacts with experts in assay development. Join our speed networking session to meet a variety of attendees at the summit, and exchange details to catch up later.
Adopting Technological Innovations in Potency Assays to Improve Testing Efficiency & Accuracy
11:30 am Leveraging AI for Enhanced Potency Assay Development in Cell Therapies
Synopsis
- Identifying AI-driven solutions to address bottlenecks in current assay development workflows that hinder effective potency measurement
- Exploring AI methodologies to optimize assay design and execution for improved consistency and reliability
- Engaging audience participation through interactive discussions and hands-on activities to apply AI concepts in real-world assay development scenarios
12:30 pm Integrating New Technologies for Cell Potency Assurance
Synopsis
- Evaluate a new instrument combining imaging and impedance for assessing T-cell potency
- Discuss how improving cell health during gene editing can enhance the final product’s potency
- Highlight the impact of early process decisions and collaborations with larger pharma partners on potency assurance
1:00 pm Panel Discussion: Maximizing Efficiency Through Closed Systems & Automation to Enhance Compliance & Reduce Operational Risks
Synopsis
- Investigating the role of closed systems in streamlining potency testing
- Comparing automated workflows for improving compliance and batch reproducibility
- Assessing how automation enhances potency assay efficiency across development stages
1:30 pm Lunch Break & Networking
Navigating Regulatory Challenges & Reducing Batch Variability to Ensure Compliance & Smooth Approvals
2:30 pm Leveraging NGS for Accurate Characterization of Reagents in Potency Assays
Synopsis
- Highlighting the role of NGS in verifying the accuracy of vector sequences and preventing mutations
- Emphasizing the importance of NGS in ensuring the safety and efficacy of cell therapy products by checking the accuracy of CAR sequences
- Discussing the development of Takeda’s in-house NGS platform resulting in significant cost savings and improved control over sequencing processes
3:00 pm Trends in Measurements Used as Potency Tests for Cell Therapy Products
Synopsis
- The mean number of potency tests per CTP for the 28 US FDA approved CTPs is 3.5 (SD = 2.0)
- 52% of the non-redacted potency tests (36 out of 67) were measurements of cell viability and cell count
- 57% of CTPs (16 of 28) reported both measurements of cell viability and count and measurements of gene and protein expression as potency tests
3:30 pm Afternoon Refreshments & Networking
4:00 pm Navigating Regulatory Expectations for Demonstrating Potency of a Phase 1 Allogeneic Product
Synopsis
- FDA recommendations and approaches for potency/functional assessments of investigational cell therapy products
- Differences in FDA and EMA regulatory expectationsÂ
- Considerations for surrogate assays and cell-based potency/functional assay developmentÂ
4:30 pm Panel Discussion: Regulatory Considerations on Potency Assays for Decision-Making During Development
Synopsis
- Early phase vs. late phase regulatory expectations for cell-based products
- Challenges in the regulatory frameworks for CGT potency assays
- Potency assurance considerations with respect to cell therapies
Moderator: Diana Colleluori