8:00 am Coffee & Networking

9:10 am Chair’s Opening Remarks

Exploring the Potential of Base & Prime Editing Technologies

9:15 am Safety of CRISPR-Cas9 in Therapy: A Call to Action

Synopsis

  • CRISPR-Cas9 overview
  • News from the field
  • Consequences of Double strand breakage:
  • OFF-target editing: how to detect them
  • ON-target toxicity: tools to assess it
  • Tumorigenicity assays
  • Conclusions

9:45 am Compact Adenine Base Editor with Novel Microbial Components

Synopsis

• More details TBC

10:15 am Exploring the Potential of Prime Editing

Synopsis

• Discuss prime editing systems for introducing precise genome edits, and their potential as therapeutics for addressing diverse genetic disorders
• Examine data demonstrating the capability of prime editing systems to mediate both small and large deletions and insertions
• Explore recent developments in prime editing systems

10:45 am Morning Refreshments & Networking

11:35 am Singling Out the Cuts: Single-Cell Multi-Modal Analysis for CRISPR- Edited Cells

Synopsis

  • Editing cells with CRISPR results in cell populations that have cell-to-cell variation in editing outcomes
  • Currently, time-consuming clonal isolation and expansion is necessary to identify the editing profile of individual cells
  • With the Tapestri Platform, it is possible to measure multiplex edits, zygosity, and off-target events across thousands of cells in a single assay
  • This powerful technology holds much promise for modelling diseases like cancer, and to characterize cell and gene therapies

11:45 am EMD-101, A Potential Autologous HSC-Based Therapy for ELANE-Related Severe Congenital Neutropenia

  • Rafi Emmanuel Senior Vice President, R&D, Emendo Biotherapeutics

Synopsis

  • EMD-101 is a patient-derived HSCs product with a specific knock-out of ELANE mutated allele, that reverts the block of myeloid differentiation, typical of severe congenital neutropenia (SCN)
  • The editing profile of our product undergoes a thorough genomic analysis to meet the highest safety standards
  • Our product maintains the core functions of neutrophils in vitro, in addition to efficient engraftment and multi-lineage differentiation in vivo

Explore Promising CRISPR & Genome Editing Preclinical & Clinical Studies

12:15 pm Innovating Xenotransplantation with Genome Editing

  • Jacob Layer Senior Scientist, Group Leader, eGenesisbio

Synopsis

• Xenotransplantation history and challenges
• Genome editing and xenotransplantation
• Development of genome edited organs for xenotransplant

12:45 pm Function & Discoveries of the Arbor Discovery Line Using Type V Systems

  • Roy Ziblat Scientific Manager, Arbor Biotechnologies

Synopsis

• Testing and characterization of novel systems
• Arbor discovery process and throughput
• Nuclease characterization
• Target portfolio
• Transposases discovery

1:15 pm Lunch & Networking

2:15 pm SEMMs: Somatically Engineered Mouse Models, A New Tool for Translational Preclinical Research

Synopsis

• Somatically Engineered Mouse Models (SEMMs) with the CRISPR/Cas9 system have optimized and made more agile mouse modelling
• SEMMs can be integrated in the drug discovery pipeline at different levels
• SEMMs are a versatile platform that can be used for target ID in vivo

2:45 pm Development of Systemic CRISPR-based Therapeutics

  • Sean Burns Vice President, Disease Biology, Intellia Therapeutics

Synopsis

• Systemic non-viral delivery of CRISPR/Cas9 is a safe and effective way to perform gene editing in vivo
• Platform enables potentially curative therapies with a single IV administration
• CRISPR-mediated targeted gene insertion provides durable, high-level expression of therapeutic proteins
• Non-viral delivery to multiple tissue types opens the door to new therapeutic applications

3:15 pm Investigate the Therapeutic Potential of CRISPR Technology in Treating Sickle Cell Disease

Synopsis

  •  Explore the potential to treat sickle cell disease using precision gene repair
  • Understand how this gene correction approach differs from others being investigated in sickle cell disease
  • Learn about data being used to inform clinical trial design

3:45 pm Chair’s Closing Remarks

4:00 pm Close of Summit