8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
9:00 am Industry Leader’s Panel: Overcoming Profitability, Analytical Challenges, Emerging Technologies, & Future Directions
Synopsis
- Profitability Challenges of Gene Therapies: Discussing the high cost of gene therapy production and pricing models
- Emerging Analytical Technologies: Exploring how long-read sequencing, CRISPR-based assays, and advanced vector characterization tools are improving gene therapy product quality
- Analytical Development Bottlenecks: Addressing critical challenges in gene therapy analytics, including assay scalability, batch-to-batch consistency, and limitations in viral vector characterization
9:45 am Session Reserved for BioRad
10:15 am Presentation & Roundtable Discussion: A Review of Gene Therapy Release Assays & Their Utility
Synopsis
- Are all assays used for release of AAV products truly confirming a product quality attribute?
- Are the fundamentals for these methods sound?
- Are we interpreting the data from these assays correctly?
- Is it too late to do something about this?
10:45 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
Bioassays & Molecular Biology
Enhancing Potency Assessment in Gene Therapy for Reliable Assays & Regulatory Success
Overcoming the Challenges of NGS Validation for Gene Therapy Potency & Stability to Improve Consistency
Synopsis
- Validating NGS for accurate transgene integrity and potency measurement in gene therapy, with a focus on discrepancies observed when correlating NGS results with AUC and other potency assays
- Discussing the challenge of obtaining only partial sequence coverage and how these unexpected results can complicate potency correlations, often leading to discrepancies when compared to traditional methods
- Exploring recent advancements in NGS technology and best practices to improve the reliability and consistency
12:15 pm Optimizing Potency Assays for Gene Therapy From Particle Correlation to QC-Friendly Methods
Synopsis
- Explore the correlation between full, empty, and partials and their impact on potency assays.
- Develop a roadmap for efficiently streamlining potency method development while meeting regulatory requirements and scientific needs
- Discuss when to initiate potency assay efforts, and how to decide between using early-phase assays or developing entirely new QC-friendly methods
12:45 pm Lunch Break & Networking
1:45 pm Defining Potency for LNP-Delivered Gene Therapies: From mRNA to Function
Synopsis
- Developing robust potency assays for mRNA and guide RNAbased drug substances
- Measuring functional outcomes of LNP-delivered gene writers beyond traditional AAV frameworks
- Overcoming assay challenges in transient expression and regulatory expectations
Innovative Technologies in Gene Therapy Looking to Advance Vector Characterization & Genome Integrity
2:15 pm Session Reserved for Intertek
2:45 pm Advancing Molecular Techniques for Vector Characterization in Heterogenous Capsid Populations
Synopsis
- Detailed analysis of enriched full, intermediate, and empty capsids isolated by ultracentrifugatio
- Exploring capsid-associated DNA and the potential role it plays in nuclease resistance
- Discussing DNA conformation and the need for restriction enzymes in ddPCR based on vector design and promoter selection
3:15 pm Afternoon Break & Poster Session
4:15 pm Ensuring Genome Integrity in Gene Therapy with Advanced Tools & Techniques
Synopsis
- Overview of molecular biology tools for analyzing the structure and integrity of vector DNA in gene therapy products
- Exploring high-throughput methods to evaluate vector DNA and detect potential structural issues during the gene therapy production process
- Discussing techniques to assess proper gene integration, minimize off-target effects, and ensure genomic stability in gene-edited therapies
4:45 pm Ensuring Consistency in Measured DNA Concentration while Transitioning from qPCR to Advanced PCR Methods
Synopsis
- Selecting the appropriate tool fit for your research to identify DNA-related Critical Quality Attributes (CQAs) by comprehending the current requirements
- Addressing methodological differences between the techniques to ensure consistency in results across these platforms
- Key factors that regulatory agencies focus on when validating assay methods, and how to demonstrate that different techniques are consistent for reliable product release testing
Physicochemical Properties
Advancing Analytical Methods for Complex Gene Therapy Products
11:45 am Making AUC GMP-Ready: Optimizing for QC & Regulatory Use
Synopsis
- Streamlining AUC for QC: Improving robustness and simplifying data workflows for consistent results
- Validation & Compliance: Meeting regulatory expectations for method validation and system suitability
- GMP Integration: Using AUC to support full/empty analysis in release and stability testing
12:15 pm Aggregation Analysis in AAV: Selecting Fit-forPurpose Assays
Synopsis
- Strengths and limitations of DLS, SEC-MALS, and AUC for size and aggregate profiling
- Choosing methods based on capsid type, formulation, and aggregation liability
- Defining actionable aggregation limits that align with product quality and safety
12:45 pm Lunch Break & Networking
1:45 pm Shining Light on Mass Photometry: Applications & Analytical Challenges in Gene Therapy
Synopsis
- Explore how mass photometry enables rapid, label-free analysis of viral vector heterogeneity, distinguishing between full, empty, and partially filled capsids with high sensitivity
- Discuss how this technique supports early-stage development and QC workflows, especially when sample availability is limited and rapid results are needed
- Address current hurdles such as sensitivity to buffer conditions, lack of standardized protocols, and the path toward integrating mass photometry into GMP-compliant environments
Defining & Controlling Product Quality to Ensure Safety, Consistency, & Compliance
2:15 pm Session Reserved for Mirus Bio
2:45 pm Roundtable: Setting Meaningful Specifications for AAV Aggregates & Particles in Commercial Readiness
Synopsis
- Defining Release Criteria for Commercialization: Establishing clear thresholds for full, empty, and partial particles to meet commercial product release standard
- Control Strategies for Intermediate Particles: Implementing robust analytical strategies to monitor intermediate particles and ensure consistency in commercial production
- Linking Particle Profiles to Commercial Outcomes: Understanding how particle distribution impacts potency, safety, and long-term product performance in the market
3:15 pm Afternoon Break & Poster Session
4:15 pm Aging Like Fine Champagne: Ensuring Consistent Quality with Reference Standards
Synopsis
- Just as a fine champagne maker relies on multiple vintages to achieve consistent quality, manufacturers must employ both primary and working reference standards to ensure product consistency throughout its lifecycle
- Highlight the working reference standard’s role in bridging gaps when the primary standard no longer reflects the product’s original characteristics, ensuring uninterrupted quality assurance
- Explore how planned transitions from primary to working reference standards, supported by stability testing, maintain long-term product quality and compliance, much like ensuring consistent champagne flavor over decades
4:45 pm Establishing Infectious Titer Assays with Accuracy & Confidence
Synopsis
- Infectious Dose vs. Genome Count: Understanding the gap between total vector genomes and actual infectious units
- Reducing Variability: Using reference standards and optimized protocols to improve assay consistency
- Enhancing Biological Relevance: Emerging tools and approaches for more predictive and meaningful titer assays
5:15 pm Chair’s Closing Remarks
5:20 pm End of Conference Day One