8:00 am Registration & Coffee
8:25 am Chair’s Opening Remarks
FDA & Regulatory Considerations for CRISPR & Editing Technologies
8:30 am FDA’s Regulatory Approach to CRISPR Technology
Synopsis
• FDA regulates human gene therapy in the United States
• CRISPR offers the opportunity to transform disease management
• Use of CRISPR constructs may also shift the focus of development of the current generation of gene therapy vectors
• FDA will continue to work with sponsors to expedite the development of novel cell and gene therapies
9:00 am Panel Discussion: Driving CRISPR Technology to the Clinic
Synopsis
• Safety considerations are limiting the use of CRISPR in the clinic
• Identifying suitable patient groups for study
• Addressing challenges with predictability & reproducibility of genome editing
9:30 am CRISPR Without the Cut: Introducing Horizon’s CRISPRi Portfolio
Synopsis
- Data from the development of Horizon’s proprietary dCas9-SALL1-SDS3 repressor
- Use of dCas9-SALL1-SDS3 mRNA and synthetic sgRNAs for potent, reversible target gene repression
- Use of synthetic, arrayed format CRISPRi reagents to study complex readouts not conducive to pooled studies
10:00 am Morning Refreshments & Speed Networking
Evaluating & Overcoming Challenges with CRISPR Delivery Methods
10:50 am Solving the Challenges of Editing & In Vivo Delivery
Synopsis
• Engineering of natural CRISPR systems can hone therapeutic characteristics
• CasX based systems allow for efficient use of AAV
• Holistic re-engineering of natural enzymes paves a way to treat diseases with significant unmet need
11:20 am Expanding the Genome Editing Toolbox with Metagenomics
Synopsis
- Through metagenomics, reconstructed genomes from unknown organisms are searched to discover novel nucleases of all types
- High throughput characterization processes enable rapid development of effective and precise gene editing tools with minimal engineering required
- Uncovering next-generation technologies: novel CRISPR nucleases, base editors, and transposases
11:50 am CRISPR-Associated Nucleases for Gene Editing: Tools to Support Discovery and Therapeutic Programs
12:00 pm Lunch & Networking
1:30 pm New Delivery Vehicles for Gene Editing Enzymes
Synopsis
• Delivery vehicles for the Cas9 RNP
• Gene editing after a direct tissue injection
• New linkers for conjugating proteins to polymers
• Gene editing in organs outside of the liver
2:00 pm CRISPR Delivery via the Use of Lipid Nanoparticles Delivery
Synopsis
• The state of the LNP field
• High-throughput LNP barcoding for mRNA delivery
• Novel LNPs for non-liver delivery
2:30 pm Afternoon Refreshments & Poster Session
Identify Strategies to Improve Safety & Reliability of CRISPR to Advance Clinical Acceptance
3:30 pm Novel CRISPR-Associated Gene-Editing Systems Discovered in Metagenomic Samples Enable Efficient & Specific Genome Engineering For Cell Therapy Development
Synopsis
• Explore the discovery of new CRISPR enzymes from metagenomic data, both type II and type V systems and discuss & compare systems
• Investigate high-level editing in primary T cells at the alpha-chain of the TCR locus (key locus for allogenic CAR T development)
• Simultaneous inactivation of the beta-chain TCR locus enables TCRbased T cell therapeutics dev
• Investigate the translatability of both internal and CMO-based protein manufacturing established
4:00 pm Useful Engineered CRISPR-Cas Enzymes with Enhanced Properties
Synopsis
• Protein engineering enables the development of genome editing technologies with improved properties
• Exploration of CRISPR-Cas enzymes for new biotechnology applications
• Engineered CRISPR-Cas variants for therapeutic applications
4:30 pm Arbor Discovery’s Novel Process and Protein Engineering Approach
Synopsis
• Nuclease portfolio
• Editing capabilities and specificity
• Therapeutic applications
5:00 pm Chair’s Closing Remarks
5:15 pm Close of Day 1
