DAY TWO - Thursday | May 29, 2025
7:30 am Registration & Coffee
7:55 am Chair’s Opening Remarks
08:00 – 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.
10:00 am Morning Refreshments
Optimizing Cell Line Engineering to Produce More Replicable & Accurate Potency Assays
11:00 am Advances in Generating Effective Cell Lines for Potency Assays
Synopsis
- Techniques for cell line development to improve efficiency
- Selecting cell lines for specific assays to enhance relevance
- Ensuring cell line stability and performance for better outcomes
11:30 am Use of New Techniques & Technologies in Cell Line Engineering
Synopsis
- CRISPR and other gene-editing technologies to enhance precision
- High-throughput cell line screening to increase efficiency
- How can automation in cell line development reduce costs?
12:00 pm Lunch Break
1:00 pm From In Vivo to In Vitro: Harnessing Cell Line Engineering for Potency Assay Optimisation
Synopsis
- Enhancing Transduction Efficiency: Increased transduction and reduced assay background to enhance sensitivity and dynamic range.
- Overcoming Tissue-Specific Promoter Challenges: Tackling cell line limitations caused by tissue-specific promoters
- Minimizing Variability with Thaw-and-Use Cells: Streamlining workflows and reducing assay variability for consistent and reproducible results
Designing Potency Assays for Novel Gene Therapy Modalities>
1:30 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
2:00 pm Considerations for Potency Assay Development for Gene Editing Products
Synopsis
- Overview of matrix approach for potency assay development
- Review of available regulatory guidance and expectations for a phased-based approach to potency assay development
- Tools and considerations when measuring biological cascade of RNA & LNP delivery in-vitro
- Challenges associated with potency assay development for gene editing products
2:30 pm Afternoon Break & Structured Networking
Emerging Technologies in Assay Development that will Catalyze the Future of Gene Therapy>
3:00 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
3:30 pm Panel Discussion: Future Directions & Trends in Potency Assays
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?