7:00 am Check-In & Coffee
7:50 am Chair’s Opening Remarks
8:00 am Industry Leader’s Panel: Overcoming Profitability, Analytical Challenges, Emerging Technologies, & Future Directions
8:45 am Precision in Every Droplet: Advancing Analytics with ddPCR Technology and the QX700 ddPCR Systems
Synopsis
- Join us to explore the transformative role of Droplet Digital PCR (ddPCR) in gene therapy analytics, past present and future
- Learn about the NEW Vericheck ddPCR Empty-Full Capsid Kit, enabling scalable and reliable calls of your AAV empty, full, and partial capsids
- Meet the QX700 ddPCR System—Bio-Rad’s NEW all-in-one platform designed for advanced and scalable 7-color testing.
9:15 am Presentation & Roundtable Discussion: A Review of Gene Therapy Release Assays & Their Utility
9:45 am Morning Break & Speed Networking
Synopsis
As this community re-unites for the 7th time, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections
Enhancing Potency Assessment in Gene Therapy for Reliable Assays & Regulatory Success
10:45 am Overcoming the Challenges of NGS Validation for Gene Therapy Potency & Stability to Improve Consistency
11:10 am Optimizing Potency Assays for Gene Therapy From Particle Correlation to QC-Friendly Methods
11:35 am Low-variability Titer, VCN, and Genome Integrity Quantification with Countable PCR
11:45 am Defining Potency for LNP-Delivered Gene Therapies: From mRNA to Function
12:10 pm Lunch Break & Networking
Innovative Technologies in Gene Therapy Looking to Advance Vector Characterization & Genome Integrity
1:10 pm Developing Effective Analytical Strategies for In-Depth Full vs Empty Capsid Characterization
Synopsis
- Why Full versus empty capsid quantification is important.
- What are the recommended orthogonal techniques available>
- Summary of case study based on AAV 8 products using Cryo-TEM, Analytical Ultracentrifugation (AUC), Capillary Electrophoresis (CE), SEC-MALS, MADLS, and ddPCR. Each method provided complementary insights into capsid composition, genome content, and particle integrity
1:40 pm Ensuring Genome Integrity in Gene Therapy with Advanced Tools & Techniques
2:05 pm A case study for bridging qPCR assay to ddPCR for quantifying residual plasmid in AAV
2:55 pm Afternoon Break & Poster Session
Advancing Analytical Methods for Complex Gene Therapy Products
3:40 pm Session Reserved for Millipore Sigma
4:05 pm Session Reserved for Mirus Bio
4:10 pm Aggregation Analysis in AAV: Selecting Fit-for-Purpose Assays
4:35 pm Shining Light on Mass Photometry: Applications & Analytical Challenges in Gene Therapy
5:25 pm Chair’s Closing Remarks
5:30 pm End of Conference Day One