8:00 am Check-In & Coffee
Early-Stage Development
WORKSHOP A
9:00 am Titer & Potency Strategies for IND Filing – Building a Robust Analytical Foundation for Regulatory Success
Synopsis
As gene therapy programs move rapidly toward IND/IMPD submissions, ensuring the right titer and potency assays are in place from the earliest stages is critical. This workshop will dive into essential early-stage assay design and long-term planning considerations to meet evolving regulatory expectations. Attendees will explore how to prioritize analytical methods, ensure assay suitability through qualification or validation, and build a lifecycle approach to support development through to commercialization.
Key discussion points:
- Defining Essential Early-Stage Assays: Establishing the core titer and potency assays needed to support IND/ IMPD submissions and ensure alignment with regulatory expectations
- Analytical Lifecycle Planning: Clarifying when to qualify vs. validate assays during development, and how to avoid rework during late-stage filings
- Future-Proofing for Regulatory Compliance: Building assay strategies that scale with your program, from early discovery through to pivotal trials and BLA submission
Late-Stage Development
WORKSHOP B
9:00 am Advanced Stability Testing: Implementing Stress & Accelerated Stability Conditions for Commercial Readiness
Synopsis
Establishing robust stability profiles is essential yet challenging due to the limited understanding of degradation pathways and assay limitations. This session will focus on designing effective stress and accelerated stability studies, interpreting degradation data to support shelf-life and retest justifications, and navigating evolving regulatory expectations. Attendees will explore best practices for building confidence in product stability.
Key discussion points:
- Designing Meaningful Forced Degradation & Stress Studies: Share approaches to stress testing (thermal, agitation, oxidative, freeze-thaw) and how data informs product understanding and predictive modeling
- Selecting and Validating Stability-Indicating Assays: Discuss the assays best suited to monitor relevant CQAs, challenges in detecting subtle degradation, and how to ensure assay sensitivity and robustness
- Shelf-Life Justification & Regulatory Strategy: Explore how companies justify retest dates, handle data variability across sites, and build regulatory packages that withstand scrutiny for commercial approval
12:00 pm Lunch Break
WORKSHOP C
1:00 pm Ensuring Genome Integrity: Analytical Strategies for CRISPR-Based Editing
Synopsis
As CRISPR/Cas technologies become central to advanced therapies, developers must create robust, sensitive assays to assess editing efficiency, off-target effects, and genomic integrity. This session explores evolving tools and best practices to ensure precise gene editing events that are QC-ready.
Key Discussion Points:
- Designing the Right Assays: Compare the strengths and limitations of NGS, ddPCR, and other platforms for quantifying editing efficiency and detecting rare off-target events
- Evaluating Genome Integrity Beyond the Edit Site: Address strategies for detecting large structural changes like translocations, deletions, and potential genome imprinting impacts, which may affect gene expression and therapeutic efficacy
- Optimizing for Scalability and Standardization: Key considerations for adapting assays for QC, transfer to release testing, and ensuring scalability in clinical applications
WORKSHOP D
1:00 pm BLA Submission & PPQ Strategies: Navigating the Critical Path to Regulatory Approval
Synopsis
Gene therapy products present unique challenges in analytical development. This workshop will provide practical strategies for addressing key aspects of BLA submissions, including viral vector characterization, comparability studies, and PPQ. Attendees will gain insights into ensuring product consistency, regulatory compliance, and a smooth path to approval.
Key Discussion Points:
- Regulatory Requirements for BLA Submissions: Key analytical aspects for gene therapy products, including vector characterization and potency testing
- Comparability Studies for Gene Therapy: Strategies for designing studies to ensure consistency between clinical and commercial products
- PPQ Strategies for Analytical Methods: Best practices for validating assays and ensuring consistency across production scales
4:00 pm End of Pre-Conference Workshop Day