8:30 am Chair’s Opening Remarks


Review of yesterday, and introduce the theme of the day: Expanding the CRISPR toolbox and overcoming the current limitations within functional genomics

How Functional Genomics and Screening Applications of CRISPR have Revolutionized the Drug Discovery Pipeline


How can optimizing screening techniques ensure greater clinical success? Technological innovations within screening and improved target identification are allowing CRISPR to span in a multitude of promising directions and the amalgamation of CRISPR screening with artificial intelligence sets to transform the road towards precision personalized medicine.

8:40 am How CRISPR Based Screening Systems can be used in Parallel for Target Identification and Validation


• Description of types of genome-wide screens used for drug discovery
• Interpreting data and determining mechanisms of disease pathology from pooled CRISPR screens
• High-throughput validation of druggable targets

9:10 am CRISPR Screening on Primary T cells


• Successful demonstration of pooled lentiviral screening in primary human T cells
• Drug-gene interaction confirms anticipated responses and identifies novel targets
• Donor hit calling is evident, but key hit calling is very consistent

9:40 am New Frontiers for Pooled Screens: Finding Regulatory Elements in the Noncoding Genome and Capturing Multi-Cell Interactions

  • Neville Sanjana Core Faculty Member and Assistant Professor, New York Genome Center and Departments of Biology and Neuroscience


• High-throughput methods to perturb genes and noncoding elements in the genome
• Interpreting the data from designing and introducing a pooled CRISPR library
• Identifying novel genetic mechanisms for cancer immunotherapy

10:10 am Combining CRISPR Editing and Cell Barcoding to Elucidate Gene Function and Advance Target Discovery


– Labeling individual cells with unique barcodes detectable by both NGS and RNA sequencing provides a way to identify cell subpopulations and track fractions of cells experiencing phenotypic changes such as activation, differentiation, etc

– Cellular barcoding with genetic effector libraries, such as CRISPR sgRNA libraries, can be used to tease out the phenotypic changes and response from biological perturbations in cell subpopulations

– Robust gene expression profiling of many thousands of cells requires a targeted RNA sequencing approach compatible with barcoding techniques


10:25 am Morning Refreshments

10:55 am Synergy between CRISPR Functional Genomics and Human Genetics to Enhance Biopharmaceutical R&D Pipeline Success


• Building CRISPR functional genomics infrastructure at AbbVie
• Use of in vitro and in vivo CRISPR screens to drive novel target discovery and translational research
• CRISPR approaches to functional validation of human genetics data

11:25 am How to Build a Streamlined Approach to CRISPR Screening

  • Ultan McDermott MB PhD Chief Scientist and Former Group Leader, Wellcome Sanger Institute, AstraZeneca, Honorary Faculty


• Using different screening libraries to build high quality combined datasets
• Strategies for the analysis of genome-scale CRISPR data, from classical statistics to artificial intelligence
• How to create a ‘useful’ computational pipeline – finding needles in haystacks
• Efficient validation protocols post genome-wide CRISPR screens

11:55 am Discovering and developing next-generation CRISPR platforms for diagnostics


• Newly discovered CRISPR systems contain unique properties that improve the CRISPR toolbox
• How we translate pioneering research to build a CRISPR platform
• Exploration of the initial platform, how it makes use of an aresenal of unique CRISPR proteins and how they allow us to sense virtually any type of nucleic acid

12:25 pm Arrayed and pooled CRISPR screening in a complex primary cell model of the human airway


  • Establishing highly efficient genome editing in primary human bronchial epithelial cells (HBECs) differentiated at air liquid interface (ALI) – for arrayed screening.
  • Cell model and assay validation for pooled CRISPR screening.
  • Pooled CRISPR screening in primary HBECs differentiated at ALI and hit validation.

12:55 pm Networking Lunch

Progression within the Vexing Dilemma of In-Vivo Delivery


Delivery is the limiting factor for the progression of CRISPR-based genetic engineering. What’s the point in having all these precision tools if we can’t yet confidently utilize them for in-vivo applications? Historically, adeno-associated virus vectors have been the adopted delivery method, but long expression of Cas9 and complicated targeting issues has led to a shift in focus. Here, we explore innovative methods for in-vivo delivery including the use of nanoparticles.

2:00 pm Viral Engineering Strategies to Alleviate Barriers Towards Successful Delivery: A Specific Focus on the Retina


• The use of AAV vectors for gene delivery
• Gene therapy & gene editing in retina
• Engineering a way to reduce the temporal expression of Cas9

2:30 pm Exploring Innovative Methods for Non-Viral In-Vivo Delivery


• How to use nanoparticles for more effective delivery
• How this delivery technique overcomes previous in-vivo hurdles
• Potential applications for advanced therapy
• Update as to where we are in the field currently

Generating More Predictable Disease Models with CRISPR


As we aim to understand the backbone of genetic diseases and seek to discover treatments, the use of genetic models for diseases is vital. Here we aim to extrapolate shared learnings when it comes to CRISPR editing on different cell types and how to design a pipeline for CRISPR editing and iPSC disease modeling

3:00 pm CRISPRing Made Easier to Create Animal Models for Basic and Drug Discovery Research

  • Guru Channabasavaiah Associate Professor- Genetics, Director- Mouse Genome Engineering Core Facility, University of Nebraska Medical Center


• Introduction to decades-old traditional transgenic technologies to create animal models
• Discussion of technological breakthroughs such as Easi (Efficient additions with ssDNA inserts)-CRISPR and Genome editing via Oviductal Nucleic Acids Delivery (GONAD)
• Discussion of how the latest technological advances have redefined the traditional transgenic methods in CRISPRing animal genomes

3:30 pm Generation of humanized mouse models

  • Wojtek Auerbach ISTT Presdent, Senior Director Emeritus, Embryonic Stem Cells Technologies, Regeneron Pharmaceuticals Inc.


• To test drug candidates we produce mice expressing a gene for human target
• We replace entire mouse gene loci with their human orthologue
• Our approach consists of simultaneous electroporation of ES cells with RNPs targeting a mouse gene to be deleted with a human BAC-based targeting vectors

4:00 pm Industrializing iPSC CRISPR Editing and Disease Modeling


• An industry perspective of using CRISPR and iPSCs
• How to design a pipeline for CRISPR editing and iPSC disease modeling
• How Artificial Intelligence can improve iPSC cultures

Thinking ahead: Standardization of CRISPR Protocols and Commercialization


Eliminating reproducibility issues by introducing CRISPR protocols. Discussions based on the commercialization of CRISPR seem rapid, but it’s worth looking forward considering the short period of time taken to race CRISPR to the clinic.

4:30 pm Panel Discussion: Standardization and Reproducibility of CRISPR Protocols Across the Industry

  • Dan Tierno Associate Director, Global Clinical Data Sciences and Analytics, Bayer
  • Samantha Maragh Leader, Genome Editing Program, NIST


• What standards should be set to efficiently drive the translation of genome editing to the clinic
• How can we endeavour to co-create protocols for best practice with normalized data
• Harmonizing global regulatory bodies

5:20 pm Chairs Closing Remarks

5:30 pm End of Day Two & Close of 5th Precision Genome Editing Congress 2019