Conference Day One
Wednesday, March 12, 2025
7:30 am Check-In, Coffee & Light Breakfast
8:00 am Chair’s Opening Remarks
Optimizing Potency Assay Development Strategies for Enhanced Assay Robustness & Validity
8:30 am Evaluating Early-Stage Development Considerations to Build a Strong Foundation for Assay Success and Efficient Transfer
Synopsis
- Assessing Key Factors in Designing and Developing New Potency Assays
- Identifying Initial Challenges in Assay Development within the CAR-T Space
- Overcoming Challenges in Assay Transfer from Development to Clinical/Commercial Stages While Ensuring Consistency and Regulatory Compliance
9:00 am Advancing Pivotal Clinical Trials: Strategic & Technical Insights for Assay Development
Synopsis
- Transitioning from early-stage to pivotal trials and leveraging proprietary editing techniques to ensure robust and reliable assays for clinical success
- Planning potency assays, evaluating parameters and regulatory impacts for accuracy and compliance
- Sharing data and case studies, addressing challenges to accelerate development and improve efficiency
9:30 am Refining Late-Stage Assay Validation to Ensure Regulatory Approval
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 Morning Refreshments & Speed Networking
Adopting Technological Innovations in Potency Assays to Improve Testing Efficiency & Accuracy
11:30 am Advancing New Technologies for Assay Quantification to Improve Accuracy & Accelerate Development Timelines
Synopsis
- Identifying technologies enhancing accuracy and reliability in potency quantification
- Evaluating benefits of impedance assays compared to traditional assays
- Outlining how new technologies streamline potency measures for robust validation
12:00 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
12:30 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
1:00 pm Lunch Break
Improving Raw Material Qualification & Reducing Batch Variability to Enhance Assay Consistency
2:00 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
2:30 pm Characterizing Non-Viral Gene Editing Reagents for Potency Assays
Synopsis
- Utilizing PCR, sequencing, and flow cytometry to characterize gene editing products and assess their potency
- Evaluating the impact of raw materials on the reliability of potency assay results
- Implementing methods to mitigate risks associated with raw material inconsistencies, ensuring batch-to-batch consistency of critical reagents
3:00 pm Afternoon Refreshments & Networking
Navigating Regulatory Challenges & Global Standards to Ensure Compliance & Smooth Approvals
3:30 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
4:00 pm Beyond Understanding – How Can Cell Therapy Drug Developers Implement New FDA Guidelines?
Synopsis
- Investigating the implications of the FDA’s new potency guidance for cell therapies
- Determining strategies to ensure compliance with evolving potency requirements
- Clarifying the FDA’s emphasis on Critical Quality Attributes (CQAs) for potency assurance