8:00 am Coffee & Networking

9:00 am Chair’s Opening Remarks

Exploring the Potential of Base & Prime Editing Technologies

9:15 am Evaluating the Main Safety Considerations of Base Editing

Synopsis

• The Cas9 nuclease induces a double-stranded DNA break (DSB) which may result in mis-repair events with stochastic nucleotide insertions and deletions (indels) at the target site
• Base Editors were presented as novel and safer gene editors lacking the ability to induce DSB
• Their use in therapy is still hampered by a reported gRNA-independent Off-Target editing effect at RNA level
• As of today, Off-Target editing of mRNA has been demonstrated but no other unwanted deamination has yet been showed as well as the consequences of such an uncontrolled event
• We shed light on all the dark aspects of BE safety and we prove that a more pragmatic and analytic approach is needed in order to progress these machinery to the clinics

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

11:45 am Panel Discussion: Paving the Way for Personalized Gene Edited Medicine

Synopsis

• What will personalized medicine look like?
• What factors have prohibited a shift towards personalized medicine?
• Assessing the potential of single-cell sequencing to make precision medicine more accurate
• Exploring the identification of pathogenic variants in cancer genes using base editing screens

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 Preclinical Platform for CRISPR-Mediated Gene Editing

  • Triparna Sen Assistant Attending, Memorial Sloan Kettering Cancer Center

Synopsis

• CRISPR screen
• In vivo CRISPR targeting
• Lessons in lung cancer

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 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

4:15 pm Chair’s Closing Remarks

4:30 pm Close of Summit