9:20 am Chair’s Opening Remarks
Advanced Analytics for Emerging Genome Editing Technologies to Optimize Therapeutic Strategies
9:30 am Development & Analytical Strategies for a Novel Editing Technology
Synopsis
- Discussing the development and potential applications of CRISPR-based epigenetic editing technologies, including the EPI 321 program
- Detailing the creation and validation of analytical assays for epigenetic editing, focusing on off-target and on-target analysis, potency assays, and delivery modalities
- Sharing insights on the journey from initial research and development to achieving IND and CTA approvals, highlighting key challenges and successful strategies
10:00 am Metabolic Engineering & Analytical Development of CAR-NK by CLICK, an Efficient Non-Viral CAR Integration on CISH Locus
Synopsis
- Precise CAR integration at the CISH locus enables targeted, stable, and efficient gene editing
- Non-viral CLICK platform supports analytical development of off-target profiling and integration efficiency
- CLICK CAR-NK cells show strong potency, persistence, and reduced exhaustion in vivo
10:30 am Morning Refreshments & Poster Session
Ensuring Tech Transfer to Quality Control & External Teams is Streamlined & Communicated Effectively
11:30 am Expert Discussion: Ensuring Quality Control in Complex Systems for Improved Product Quality
Synopsis
- Tackling the complexity of CMC manufacturing and QC/QA release for key components, enhancing product quality
- Addressing the analytical burden of multi-component systems and ensuring high fidelity and uniformity, improving consistency
- Implementing effective QC strategies to manage supply chain challenges and stability issues, optimizing reliability
12:00 pm Roundtable: Developing a Phase-Appropriate Release & Stability Testing Strategy
Synopsis
- Choosing appropriate assays and specifications for release and stability testing for each of your CQAs
- Establishing structure-function studies to supplement regular release panel tests
- Evolving your testing methods as you progress into and through the clinic
1:00 pm Lunch
Characterizing & Quantifying Heterogeneity in Genome Editing to Ensure Consistency
2:00 pm Expert Discussion: Leveraging Machine Learning & Artificial Intelligence for Improved Precision
Synopsis
- Applying machine learning methods to predict off-target effects with greater accuracy, enhancing safety
- Creating genome editors using advanced AI techniques, improving editing precision
- Addressing computational challenges in quantifying complex technological landscapes, optimizing data analysis
3:00 pm Regulatory Requirements for sgRNA Characterization in Early & Late Phase CRISPR-Edited TIL Manufacturing
Synopsis
- Utilizing NGS and molecular assays to characterize guide RNA
- Ensuring comprehensive analysis and phase appropriate implementation of analytical tools to meet regulatory requirements
- Separating and characterizing impurities within sgRNA lots to maintain eTIL quality