8:20 am Chair’s Opening Remarks

Applying CRISPR Beyond the Coding Genome

8:30 am Harnessing Novel CRISPR Tools for Genome Engineering and RNA Editing

Synopsis

  • Understanding advantages of RNA edits compared to DNA edits
  • Exploring potential applications of RNA editing, from basic research to therapeutic application
  • Evaluating where RNA editing should be favored over DNA editing

9:00 am Epigenome Editing using CRISPR to Treat Pain

  • Ana Moreno Founder & Chief Executive Officer, Navega Therapeutics

Synopsis

  • DNA-level repression of key genes responsible for pain transmission holds great therapeutic potential
  • Discussing future applications of epigenome regulation to treat pain using dCas9
  • Highlighting any epigenome-editing specific challenges and how best to address them

9:30 am Morning Refreshments & Networking Jonathan Gootenberg

10:30 am CRISPR Diagnostics: Expanding the Nucleic Acid Detection Toolbox by Harnessing Microbial Diversity

Synopsis

  • Utilizing CRISPR diagnostics to detect nucleic acids
  • Exploring natural CRISPR diversity to develop new multiplexed and point of care detection approaches
  • Understanding the possibilities of CRISPR diagnostics in infection disease, cancer, and genetic variants

11:00 am Programmable Gene Modulation

Synopsis

  • Discussing transient alternatives to gene editing for therapeutics
  • Exploring novel applications of gene editing therapeutics and diagnostics
  •  Presenting recent data on the development of safer, more precise gene editors

Implementing a Forward-Thinking Approach to Off-Target Effects to Keep Focus on Future Applications

11:30 am Broadly Useful Engineered CRISPR-Cas Enzymes with Enhanced Properties

Synopsis

  • The PAM requirement by DNA targeting CRISPR-Cas proteins constrains targeting
  • Elimination of the PAM requirement enables access to the entire genome
  •  A catalog of PAM-stringent variants improve the protect for therapeutic translation

12:00 pm Panel Discussion: Do CRISPR Tools Need to be Perfect?

  • Luis Barrera Head of Computational Sciences , Beam Therapeutics
  • Blair Madison Senior Director, Genetic Engineering, Poseida Therapeutics

Synopsis

Off-target effects are intrinsically linked to CRISPR utilization, with technologies being developed to minimize presence of off-target effects all the time. However, how much do off-target effects really matter? This panel discussion will address questions around off-targets, including:

  • Do we need to worry about off-target effects if application is not in humans?
  • Which applications of CRISPR justify the risk of both known and unknown off-target effects?
  • What critical quality attributes do regulatory agencies require for various CRISPR applications?
  • How important is controlling off-target effects compared to their mapping?
  • Is it acceptable to assume that some off-target effects are ‘safe’?
  • Could there be unknown consequences to off-target effects in non-coding regions of the genome?

12:30 pm Lunch & Networking

1:30 pm Comprehensive Identification of Base Editor Off-Targets in the Context of Therapeutically Relevant Delivery

  • Luis Barrera Head of Computational Sciences , Beam Therapeutics

Synopsis

  • Evaluation of experimental and computational methods to detect sgRNA-dependent off-target sites
  • Measurement and comparison of sgRNA-independent mutation rates across base editor versions
  • Establishing a baseline for ‘normal’ mutations to place base editor off-target effects in context
  • Using mRNA vs. plasmids as delivery modality and its impacts on off-target detection

2:00 pm Creating Novel Proteins to Optimize Efficacy and Specificity

  • Blair Madison Senior Director, Genetic Engineering, Poseida Therapeutics

Synopsis

  • Evaluating usefulness of ‘classic’ Cas9 in the clinic compared to basic research
  • What are the real advantages to using different Cas proteins?
  • Understanding how requirements for CRISPR/Cas systems are highly context dependent
  • Limiting p53 activation

Expanding the Scope of CRISPR to Target a Wider Range of Diseases

2:30 pm Applying CRISPR to Predict and Treat Cardiovascular Disease

Synopsis

  • Reducing cardiovascular disease risk by applying CRISPR to permanently reduce cholesterol levels
  • Discussing challenges associated with implementing this technology
  • Identifying mutations which make someone vulnerable to heart disease to give early preventative therapy

3:00 pm Afternoon Refreshments & Networking

3:30 pm Developing CRISPR-Based Cell Therapies Using a Proprietary Gene Editing System (GEMS)

  • Sicco Popma Founder, Chief Executive Officer & President , IO Biosciences

Synopsis

  • Developing a gene plug-and-play system applicable to a wide range of cell types
  • The advantage of a plug-and-play CRISPR gene editing system in allogeneic therapies
  • Exploring the potential to treat a wide range of diseases beyond cancers

4:00 pm Utilizing CRISPR to Grow Human Organs for Transplantation

Synopsis

  • CRISPR can be used to generate a niche inside of an animal to grow an organ for transplant
  • Requires host embryos to be non-mosaically edited
  • Multiple guide RNA’s efficiently generate non-mosaic knockout animals
  • The CRISPR-Cas9 system combined with AAV6 allows for selection of animals with non-mosaic knock-ins

4:30 pm Chair’s Closing Remarks

4:45 pm Close of Conference