7:00 am Registration, Coffee & Networking

7:50 am Chair’s Opening Remarks

  • Fyodor Urnov Scientific Director , Innovative Genomics Institute

8:00 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

8:30 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

9:00 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

9:30 am Slot Reserved for Horizon Discovery

Synopsis

  • Talk details to be confirmed

10:00 am Speed Networking & Morning Refreshments

11:00 am Utilizing CRISPR-Associated Transposons for Programmed Insertions Without Generating Double-Strand Breaks

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

11:30 am 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

11:50 am Optimizing CRISPR/Cas to Translate Efficiently into the Clinic

  • Kate Zhang Vice President, Biological Development , Editas Medicine

Synopsis

  •  Identifying the right CRISPR tools with desirable attributes
  • Preclinical development to assess safety and efficacy of CRISPR-Cpf1 for autologous cell therapy
  • AsCas12a (AsCpf1) as potent and more specific than SpCas9

12:20 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

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    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

12:50 pm Lunch & Networking

2:00 pm Slot Reserved for Aldevron

Synopsis

• Talk details to be confirmed

2:15 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

2:45 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

3:15 pm Afternoon Refreshments & Networking

Exploring Quality & Safety of CRISPR to Ease Regulatory Concerns

3:45 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

4:15 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

4:45 pm Chair’s Closing Remarks

  • Fyodor Urnov Scientific Director , Innovative Genomics Institute

5:00 pm End of Day One