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
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
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
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
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
