Conference Day One: Wednesday, September 18
8 AM Check In, Coffee & Light Breakfast
8:50 AM Chair’s Opening Remarks
Advancing Methods for On- and Off- Target Editing Nomination & Detection
9:00 am Editing Assays: Their Analysis and Use in Regulatory Filings
Synopsis
- Combining orthogonal, complementary assays
- Bioinformatics analysis to demonstrate safety
- Integration of these studies for FDA filings
9:30 am Quantifying the Effects of Human Genetic Diversity on Gene Editing Outcomes
Synopsis
- Discussing the latest research into genetic heterogeneity in the context of off-target editing
- Understanding the subsequent need for sensitive and genome-wide assays to identify them
- Incorporating genetic variation into analytical strategy
10 AM Morning Refreshments & Speed Networking
Synopsis
Our speed networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in gene therapy and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against the industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond
11:00 am How Confident Are You? Considerations for Detecting Edit Sequence and Frequency
Synopsis
- Understanding types of DNA sequence detection approaches
- Assessing the performance and limits of edit detection
- Unpacking challenges for reliable detection and quantitation of edits
11:30 am Roundtable Discussion: Standardizing Off-Target Assays
Synopsis
Roundtable discussions place a larger focus on group discussion. A moderator will introduce the session topic and attendees then split into groups to discuss a series of assigned questions. At the end, all groups report back on their discussions, and findings are collated.
- Benchmarking ongoing progress made by the NIST-FDA for standardizing off-target nomination and assay methods
- Unpicking the complexity of cell line choice and impact of genetic variations
- Sharing practical advice for effective qualification of off-target assays
12:15 PM Lunch & Networking
Unpicking the Complexities of CMC Analytical Testing
1:15 pm Analyzing Guide RNA (gRNA) Impurity Discovery & Quantification
Synopsis
- Understanding the challenges in assessing gRNA impurity levels, especially with longer guides, and implications for toxicology
- Methods for site-specifically identifying both sequence and chemical impurities
- What levels of impurities are acceptable for IND submissions?
1:45 pm Speaking Position Reserved for Aldevron
Synopsis
2:15 pm Analytical Method Lifecycle Management: A Case Study of Using IPRPHPLC to Determine RNA Concentration and Ratio
Synopsis
- Overview of analytical testing panel of RNA-LNP product
- Overview of analytical method life cycle management based on Quality-by-Design concept
- A case study of IPRP-HPLC method from pre-clinical through pivotal ready
2:45 PM Afternoon Refreshments & Networking
3:15 pm Characterization of Novel RNP Delivery Vehicles
Synopsis
- Overview of analytical panel for non-viral delivery vectors
- Particle composition and biophysical characterization
- Utilizing dPCR as a tool for particle potency and biodistribution analysis
3:45 pm Robust Potency Assay Strategies: Lessons Learned from Clinical Development
Synopsis
- Defining a potency assay approach based on the disease mechanism-of-action
- Learning from a successful potency assay strategy for IND clearance
- Sharing a case study of adapting a potency assay matrix strategy through early- to late-stage development
4:15 pm Panel Discussion: Developing a Phase-Appropriate CMC Analytical Strategy
Synopsis
- What should a specification list look like for release material into the clinic?
- How do assay qualification requirements vary depending on the stage of development?
- Ensuring phase-appropriate potency assay testing