Conference Day One: Wednesday, September 18

7:00 AM Check In, Coffee & Light Breakfast

8:00 AM Chair’s Opening Remarks

  • Carl Co Senior Director - Analytical Sciences, Arbor Biotechnologies

Advancing Orthogonal Methods for Precise & Sensitive Editing Assays

8:10 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

8:40 am Revolutionizing Gene Editing with Advanced CRISPR Analytics

  • Juan-José Ripoll CQC Sr Scientific Advisor & CIDEGENT Distinguished Investigator, CRISPR QC

Synopsis

  • Maximize gene editing efficiency with real-time kinetic insights from our breakthrough CRISPR Analytics Platform
  • Gain quantitative insights into RNP formation, DNA target binding kinetics, and cleavage activity
  • Fast-track your breakthroughs by leveraging our insight solutions

9:10 am Quantifying the Effects of Human Genetic Diversity on Gene Editing Outcomes

  • Luca Pinello Associate Professor, Massachusetts General Hospital 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

9:40 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

10:40 am Role of Scalable Sequencing Tools in Attaining Precision Genome Editing

Synopsis

  • Obtaining precise outcomes from genome editing workflows relies on the ability to accurately measure both component quality as well as editing effects in cells
  • Sequencing plays an important role as the primary quality control tool for many stages of genome editing R&D and manufacturing, from vector and plasmid QC to on- and off-target assays of genome editing outcomes
  • The standardization of assays and tools that bring together flexibility, scalability and reliability for the QC of genome editing components and editing outcomes will assist in the robust and reliable development of editing technologies and human therapeutics

11:10 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

11:40 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.

This roundtable will cover:

  • 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

12:20 PM Lunch & Networking

Unpicking the Complexities of CMC Analytical Testing

1:20 pm Analyzing Guide RNA (gRNA) Impurity Discovery & Quantification

  • Eric Anderson Director, Discovery & Molecular Engineering, Vor Bio

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 recommended for IND submissions?

1:50 pm CRISPR Genome Editing Solutions from Discovery to Clinic

Synopsis

  • Aldevron – a partner for CRISPR-based therapies
  • Regulatory guidance for gene editing components
  • Assays developed to characterize RNPs

2:20 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

2:50 PM Afternoon Refreshments & Networking

3:50 pm Fireside Chat: 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

4:20 pm Panel Discussion: Developing a Phase-Appropriate CMC Analytical Strategy

  • Jie Li Director, Vertex Pharmaceuticals
  • Kok Seong Lim Senior Director - Analytical Sciences & Quality Control, Formerly of Metagenomi
  • Yuqing Chang Associate Director, 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

5:00 PM Chair’s Closing Remarks

  • Carl Co Senior Director - Analytical Sciences, Arbor Biotechnologies

5:10 PM Drinks Reception

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