Conference Day One
Wednesday, March 12, 2025

7:30 am Check-In, Coffee & Light Breakfast

8:50 am Chair’s Opening Remarks

Optimizing Potency Assay Development Strategies for Enhanced Assay Robustness & Validity

9:00 am Advancing Pivotal Clinical Trials: Strategic & Technical Insights for Assay Development

  • Kathy Simon Vice President - Global Quality, Cellectis

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

  • Shawna Organ Senior Scientist – Assay Development, BlueRock Therapeutics

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

  • Anna Brown Director Analytical Development, Poseida Therapeutics

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

  • John Yu Director - Analytical Development & Cell Process Sciences, Arcellx

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

  • Xiaoya Sun Scientist - Analytical Development - Cell Therapy, Takeda

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

  • Hui Wen Liu Principal Scientist - Assay Development, Affini-T Therapeutics

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

  • Carl Simon Biologist Professional, National Institute of Standards & Technology (NIST)

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?

  • Briti Saha Senior Scientist - Analytical Sciences & Technology, Century Therapeutics

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

4:30 pm Chair’s Closing Remarks

5:00 pm End of Conference Day One