DAY TWO - Thursday | May 29, 2025

8:00 am Registration & Coffee

8:25 am Chair’s Opening Remarks

08:30 – 10:00 | MORNING MASTERMIND

Mastering Tech Transfer to Quality Control Teams for Gene Therapy Potency Assays

Synopsis

This session offers a comprehensive deep dive into the critical process of tech transfer to Quality Control specifically for gene therapy potency assays. Attendees will explore the key steps involved in transferring assays from development to quality control, addressing common challenges, and discovering best practices to ensure a smooth transition.

The session will emphasize the importance of maintaining assay robustness and reproducibility, with techniques for validating assays during tech transfer and strategies to maintain performance and consistency. This interactive session provides a unique opportunity to gain practical insights and solutions that are not covered in the main conference sessions.

10:30 am Morning Refreshments

Optimizing Cell Line Engineering to Produce More Replicable & Accurate Potency Assays

11:30 am Cell Line Engineering for Optimal Potency

  • Rebecca Lyth Senior Scientist, Analytical Development, AviadoBio

Synopsis

  • Increasing transduction efficiency for increased assay sensitivity
  • Tackling the challenges of tissue specific promoters
  • Use of thaw and use cells to reduce assay variability

12:00 pm Developing Potency Assays for Emerging Gene Therapy Delivery Vectors

Synopsis

  • Outlining the emergence of gene therapy modalities beyond traditional AAV approaches
  • Evaluating appropriate potency assays to reflect the mode of action of new vectors
  • Case studies of novel vector approaches and their developmental hurdles

12:30 pm Lunch Break

Emerging Technologies in Assay Development that will Catalyze the Future of Gene Therapy>

1:30 pm Innovations in Assay Automation & High-Throughput Screening

Synopsis

  • Implementing automation to streamline assay processes and reduce costs
  • High-throughput screening techniques for rapid and efficient assay development
  • Integrating robotics and advanced software for enhanced assay performance

2:00 pm Panel Discussion: Future Directions & Trends in Potency Assays

  • Jie Li Director, Vertex Pharmaceuticals

Synopsis

  • Predicting future trends in potency assay development and application
  • Assessing the impact of emerging technologies on regulatory landscapes
  • How can we prepare for the next generation of potency assays?

3:00 pm Chair’s Closing Remarks & End of Conference