8:30 am Registration, Coffee & Networking

9:20 am Chair’s Opening Remarks

  • Fyodor Urnov Scientific Director , Innovative Genomics Institute

9:30 am KEYNOTE: CRISPR 2030: the Promise and the Challenge

  • Fyodor Urnov Scientific Director , Innovative Genomics Institute

Synopsis

  • Key translational challenges in CRISPR have moved beyond the fundamentals of genome editing itself
  • The next few years will bring key data from first-in-human trials that will guide technology optimization
  •  Scaling CRISPR to the public health challenge of monogenic disease is the major goal of the field for the next decade
  • Novel modes of engaging with the genome will provide essential augmentation of the clinical toolbox

Evaluating the Newest Developments in CRISPR Technology to Realize the Potential of the CRISPR Toolbox

10:00 am Exploring the Potential of Prime Editing

Synopsis

  • Development of a search-and-replace genome editing strategy (prime editing)
  • Scope of prime editing capabilities and potential for clinical applications
  • Understanding challenges associated with therapeutic application of prime editing and how to begin to overcome them

10:30 am Developing and Improving Base Editors

Synopsis

  • Deaminase domains in CBEs and ABEs can induce transcriptome-wide RNA off-target effects
  • Base editors can be engineered to reduce RNA off-targets, while maintaining efficient DNA on-target editing
  • Improvement of DNA and RNA specificity of current and future DNA base editor platforms will be important for clinical translation

11:00 am Slot Reserved for Horizon Discovery

Synopsis

  • Talk details to be confirmed

11:30 am Speed Networking & Morning Refreshments

12:30 pm Utilizing CRISPR-Associated Transposons for Programmed Insertions Without Generating Double-Strand Breaks

  • Sanne Klompe Graduate , Sternberg Lab, Columbia University

Synopsis

  • Elucidating the biological mechanism of this novel system
  • Discussing the differences between this and other CRISPR-Cas genome editing tools
  • Highlighting potential future applications of the technology

1:00 pm Functional CRISPR Screening and Cell Barcoding to Identify Genes Driving Biological Responses and Disease Progression

Synopsis

  • Pooled lentiviral libraries of CRISPR sgRNA have proven to be a highly effective approach to identify genes, and other genetic elements, driving biological responses
  • Libraries containing unique barcode sequences enable cell-specific labeling of individual cells in a target population to identify sub-populations that express specific features or phenotypes (e.g., pathway activation, drug resistance)
  •  Barcoded CRISPR guide library combined with single cell expression profiling enables parallel analysis of differences in gene activation across and within cell populations as a result of perturbation or selection

Discussing Current Clinical Applications of CRISPR to Enhance Future Gene Editing Programs

1:20 pm Precise, Non-Viral, CAR-T Engineering with CRISPR sgRNA and ssDNA

  • Lumeng Ye Senior Scientist, GenScript Biotech Corp.

Synopsis

  • T cell engineering has traditionally been done with viral vectors, but this approach has limitations including knock-in (KI) size restrictions and random insertion
  • Direct delivery of CRISPR Cas9 protein, synthetic gRNA, and donor DNA has the benefit of both precise CAR KI and simultaneous knockout (KO), and without the need for a viral vector
  • GenScript has develop several tools to efficiently manufacture sgRNA and long single-stranded DNA (ssDNA or ssODN) for precise CAR KI in T cells using a viral free method

1:50 pm Lunch & Networking

3:30 pm Slot Reserved for Aldevron

Synopsis

• Talk details to be confirmed

3:45 pm Bridging the Gap Between Academia and Industry: Translating Cutting-Edge Technologies from the Lab to the Clinic

  • Satinder Rawat Senior Licensing Officer , University of Massachusetts

Synopsis

  • Benchmarking available tools to establish the most suitable technology for a specific therapeutic goal
  • Exploring bottlenecks associated with moving new technologies from an academic to an industry setting and effectively addressing them
  • Exploring partnering models between academia and industry to accelerate development of promising technologies

4:15 pm Target Optimized Variant of CRISPR Associated Nuclease Enables Allele-Specific Knock Out of ELANE-Related Neutropenia

Synopsis

  • Our platform for optimizing the nuclease-gRNA composition enables allele-specific editing
  •  We utilize SNPs for developing allele-specific knock out strategy for a dominant negative Severe Congenital Neutropenia
  • Targeting SNPs instead of pathogenic mutations would enable higher coverage of the patients’ population with few nuclease-gRNAs combinations

4:45 pm Afternoon Refreshments & Networking

Exploring Quality & Safety of CRISPR to Ease Regulatory Concerns

5:15 pm ONE-seq: Detection of CRISPR Nuclease and Base Editor Off-Targets with Ultra-High Sensitivity to Ensure the Safety of CRISPR Therapeutics

  • Karl Petri Research Fellow , Joung Lab, Massachusetts General

Synopsis

  • ONE-seq detects CRISPR nuclease and base editor off-targets with ultra-high sensitivity
  • CRISPR off-targets need to be analyzed and avoided to clinically translate CRISPR technologies
  • ONE-seq can guide strategic decisions surrounding CRISPR safety and specificity

5:45 pm Developing Assays and Tools for Base Editing

Synopsis

  • Creating high quality reagents for base editing used for therapeutic application
  • Considering the parameters that may be required for using base editing in different indications
  •  Discussing challenges associated with gearing up for clinical trials using base editing

6:15 pm Chair’s Closing Remarks

  • Fyodor Urnov Scientific Director , Innovative Genomics Institute

6:30 pm End of Day One