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

  • TJ Cradick Chief Scientific Officer & Principal Consultant, Gene Editing Frontiers

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

  • Luca Pinello Associate Professor, Massachusetts General Hospital (MGH) and Harvard Medical School

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

  • Samantha Maragh Leader, Genome Editing Program, National Institute of Standards & Technology (NIST)

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

  • Samantha Maragh Leader, Genome Editing Program, National Institute of Standards & Technology (NIST)

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

  • Eric Anderson Director, Discovery and Molecular Engineering, Vor Biopharma

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

Aldevron: The basis for breakthroughs

2:15 pm Analytical Method Lifecycle Management: A Case Study of Using IPRPHPLC to Determine RNA Concentration and Ratio

  • Zack Zhang Director, Analytical Development, Beam Therapeutics

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 Robust Potency Assay Strategies: Lessons Learned from Clinical Development

  • Jie Li Director, Vertex Pharmaceuticals

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

3:45 pm Panel Discussion: Developing a Phase-Appropriate CMC Analytical Strategy

  • Jie Li Director, Vertex Pharmaceuticals
  • Kok Seong Lim Senior Director - Analytical Sciences & Quality Control, Metagenomi
  • Yuqing Chang Associate Director, In vivo LNP QC Lead, Beam Therapeutics

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

4:30 PM Chair’s Closing Remarks

4:30 PM End of Day One