7:30 am Registration & Networking
8:20 am Chair’s Opening Remarks
Advancing Sequencing Technologies & Automation for High-Quality, Safe CRISPR-Edited Development
8:30 am Utilizing Long-Read Sequencing & Automation to Enhance Preclinical Analytical Development
Synopsis
- Streamlining in-process testing to balance data collection with routine requirements, enhancing workflow efficiency
- Leveraging long-read sequencing to capture large fragment rearrangements, providing comprehensive structural variant analysis
- Implementing QC-friendly automation to improve efficiency and accuracy, enhancing quality control
9:00 am Advances in Next-Generation Sequencing for Precise Editing Outcomes
Synopsis
- Confirming the site and off-site integration using NGS, ensuring precise editing outcomes
- Understanding the limits of detection and quantification for NGS technologies, improving accuracy
- Scaling NGS for detecting translocations, deletions, and other non-indel events, enhancing comprehensive analysis
9:30 am Audience Discussion: Harnessing NGS for Characterizing CRISPR Components & Drug Products
Synopsis
- Applying NGS to verify sequence identity and detect impurities in CRISPR components such as synthetic guides and AAV constructs
- Ensuring product integrity and functionality through sequence-based analysis of editing efficiency and unintended modifications
10:00 am Morning Refreshments & Speed Networking
Innovating Analytics for LNPs & Novel Delivery Vehicles to Enhance Therapeutic Efficacy
11:30 am RNP Control Strategy for CRISPR-Edited TIL Manufacturing
Synopsis
- Regulatory expectations for RNP control
- Analytics for RNP characterization
- Implementation of RNP control strategy for clinical manufacture to improve gene editing efficiency and to minimize off-target editing
12:00 pm Audience Discussion: Evolving Perspectives on Delivery Vehicles & the Impact on CRISPR-Edited Therapies’ Analytical Development
Synopsis
- Assessing the performance of various novel delivery systems in delivering CRISPR components, ensuring optimal therapeutic outcomes
- Identifying and addressing the barriers to effective delivery, including immune response, off-target effects, and delivery efficiency, to enhance the precision and safety of CRISPR-edited therapies
- What are the most promising delivery vehicles currently being developed?
1:00 pm Lunch
Innovations in Computational Methods & AI to Advance Off-Target Detection & Analytical Accuracy
2:00 pm Characterizing Novel CRISPR Editors for Next-Generation Therapeutics
Synopsis
- Developing Assays for Protein Engineering and Therapeutic Development
- Novel assays for novel editors to establish data sets for regulatory filings
2:30 pm Platform Overview: Introduction to INDUCE-seq®, a PCR-free method for genome-wide mapping of double-strand DNA breaks in days, not months
Synopsis
- Therapeutic Development: Data on confirming gene editing efficiency via on-target break detection
- Applications in iPSC and T-cell therapies: Evaluation of base editor tools using INDUCE-seq®
- Preclinical Safety Assessment:
- Results from the NIST genome editing program and collaboration with HESI
- Data from studies with the Innovative Genomics Institute (IGI) supporting Gene Editing INDs
- Guide RNA Screening: Progress on SafeGuide AI, a predictive tool to differentiate safe vs. high-risk gRNA designs
3:00 pm Integrative Computational Approaches for Genome Editing for Quantification and Off-Target Characterization
Synopsis
- Presenting computational strategies for quantifying genome editing outcomes using long-read sequencing
- Exploring advanced methods for off-target prediction and variant-based characterization to improve specificity and safety in genome editing
- Bridging experimental and computational techniques to enhance detection accuracy and scalability in off-target discovery
3:30 pm Afternoon Refreshments & Networking
4:00 pm Standardizing Off-Target Analysis for Consistent Results
Synopsis
- Implementing scalable and quantifiable QC approaches for sequencing fidelity, ensuring consistent results
- Establishing standardized methods to avoid bespoke solutions for off-target analysis, enhancing reliability
- Discussing what makes the FDA confident in the off-target profile of drugs for in vivo applications, improving regulatory compliance
4:30 pm Innovating Off-Target Detection & Safety Profiles
Synopsis
- Evaluating the tolerable rates of off-target effects and their implications for safety, enhancing therapeutic safety
- Analyzing methods to quantify empty versus full particles in LNP delivery, improving delivery precision
- Integrating new technologies to enhance off-target detection and improve safety profiles, optimizing treatment outcomes