February 26-28, 2018

Boston, USA

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Day One
Monday February 26th, 2018

Day Two
Tuesday February 27th, 2018

Registration & Coffee

Chairperson’s Opening Remarks

Editing without Bounds; Realizing the Limitless Potential of CRISPR

In Vivo Genome Editing with CRISPR/Cas9 Nano Formulations


  • Advancing nanoparticle delivery for gene-editing to specific tissues
  • What is on the horizon to enhance in vivo delivery?

Engineering T Cell Circuitry


  • Cas9 ribonucleoproteins (Cas9 RNPs) enable rapid and programmable non-viral knockout and knock-in genome-editing in human T cells
  • We aim to understand how sequence variation throughout the human genome affects T cell circuits in health and disease
  • Cas9 RNP technology in combination with cell-based treatments holds great potential for therapeutic genome engineering

A Rapid, Simple, High-throughput Compatible Approach to Generating CRISPR/Cas9 Knock-out Cell Lines

  • Meiye Wu Senior Scientist, Reagent R&D, Bio-Rad Inc


  • Streamlined CRISPR/KO method that leverages ddPCR™ and HRM™ to bypass dreaded screening pain points
  • Automation compatible approach that will enable high-throughput KO cell line generation for genome-scale functional studies

Morning Refreshments

11.10 Increasing the Efficiency of the CAS HD Mechanism to Forward Your Basic Research and Drug Development

  • Overcome the Current limitations of the CRISPR technology

Guru Channabasavaiah, Director, UNMC

11.40 GenCRISPRTM Technology Makes Genome Editing Easy

  • Cases studies to show versatility of GenCRISPRTM on genome editing
  • Delivery of gRNA-Cas9 efficiently with lentivirus
  • RNP facilitate genome editing in leukemia cell lines

Leon Song, Director, Genscript

11.55 Optimizing the Activities and Specificities of CRISPR-Cas9 and Cpf1 Nucleases

  • Genome editing nucleases such as CRISPR-Cas9 and Cpf1 can be engineered to impart improved properties
  • Targeting range can be expanded by evolving variants that can target previously inaccessible sites
  • Genome-wide specificity can be improved with novel engineered variants
  • Methods to define these improved properties

Ben Kleinstiver, Instructor, Harvard Medical School

11.10 NextGEN™ CRISPR Gene Editing of Autologous and Allogeneic CAR-T Therapies

Devon Shedlock, Vice President, Preclinical Development, Poseida

11.40 Cibus’ Trait Machine is Accelerating Plan Breeding Using the Rapid Trait Development System (RTDS™) to Benefit Consumers, Farmers and Processors

  • Over thousands of years, breeders have relied on random variation for crop improvement
  • Cibus has developed a process called the Rapid Trait Development System (RTDS) that combines advanced cell culture and a range of modern mutagenesis tools to accelerate plant breeding by precisely specifying beneficial typographical changes in crop genomes much like a word processor on your computer
  • What used to be a random process taking many years can now be accomplished in months with outcomes indistinguishable from those that can occur in nature

Greg Gocal, EVP & CSO, Cibus

11.55 Translational and Basic Research-Based CRISPR Cas9 Genome Editing of Human Hematopoietic Stem Cells

  • Summarizing the current state-of-the-art CRISPR strategies to translate genome editing to the clinic for treatment of monogenic blood and immune system disorders
  • Summarizing preclinical IND-enabling studies for correcting the Glu6Val mutation in autologous HSPCs
  • Summarizing advanced CRISPR biotechnologies to interrogate HSPC gene functions

Danny Dever, Instructor, Stanford

Networking Lunch

13.40 Understanding and Manipulating the Kinetics of Cas9 Activity to Enhance Genome Editing

  • How to prospectively avoid “dud” sgRNAs for indel mutagenesis
  • Identifying the rate limiting step during Cas9-editing in cells
  • Manipulating Cas9 to provide multi-turnover nuclease activity

Brad Merill, Associate Professor, University of Illinois at Chicago

14.10 CRISPR-Cas9 Mediated Transcriptional Activation using Synthetic crRNA

  • A guide RNA platform enabling robust overexpression of genes in their endogenous context
  • CRISPR-Cas9 gene knockout and activation in onegene- per well format allows for study of biological mechanisms in complex assay

James Goldmeyer, Product Manager, Dharmacon

13.40 Future Safety and Efficacy: Exploring the Alignment of CRISPR and the Future of Therapeutic Development to the Future of Clinical Trial Design

  • Discuss how drug development and regulatory processes need to continue to embrace data science technology to enhance clinical trial efficiency for therapeutics using CRISPR as well as other innovations
  • Explore the history of the Virtual Subject model for Clinical Trial development and execution
  • Address opportunities, considerations, and best practices to drive toward the frontier of executing trials with Virtual Subjects

Dan Tierno, Strategic Implementation Manager, Bayer

14.10 Taking CRISPR to the Clini

  • Efficient editing at clinical scale and in long-term repopulating HSCs
  • Editing results in relevant pharmocodynamic effect
  • Careful choice of gRNAs results in high specificity and no detectible off-target events
  • Progressing to the clinic after scale-up and regulatory/toxicology studies

TJ Cradick, Head of Genome Editing, CRISPR Therapeutics

Afternoon Refreshments

Synthetic sgRNA Enables Highly Efficient & Consistent CRISPR Editing of Cells for Automation, Cell Engineering and Therapeutic Applications


  • Achieving consistent and high editing efficiencies with CRISPR is critical for automation, cell engineering and therapeutic applications with primary cells, and remains a significant challenge.
  • Through a collaborative effort, we demonstrate that use of synthetic sgRNA for CRISPR yields improved and consistent editing efficiencies that are required for such applications.


How do you know? Measurements and Standards to Support Confidence in Genome Editing


  • NIST is the measurement institute of the U.S., supplying unbiased expertise in measurements and standards to support growth in all sectors of economy and commerce including healthcare and the bio-economy
  • NIST has been working with experts in the genome editing field to identify precompetitive needs for establishing confidence in assessing the results of genome editing protocols
  • NIST is launching a NIST led Genome Editing Consortium as a public private partnership to bring together academia, non-profit, industry and other government to generate best practices, high quality data, a standard lexicon and data/metadata norms for the genome editing field

Genome-Scale Pooled CRISPR Screening For Drug Targets Anchored in Alzheimer’s Disease Genetics


  • Human genetic studies are being used to estimate the potential safety and efficacy of drug targets prior to pharmaceutical investment
  • Genes and pathways identified through genetic association studies are often challenging to therapeutically modulate
  • We are applying genome-scale pooled CRISPR screening to identify “druggable” regulators of Alzheimer’s disease genetic targets




Genome Editing with ZFN, Targetron, and CRISPR-Cas Systems


  • Overview of in-house genome editing innovations at MilliporeSigma
  • Surmounting limitations on CRISPR imposed by chromatin
  • Different RNA-guided options for bacterial genome editing
  • CRISPR screening tools including nuclease and transcriptional modes.

Round Table Sessions


Drive your own learning and brain storm ideas. The CRISPR speaker faculty is second to none but there is just as much knowledge in the audience as there is onstage. We want to make sure you can tap into this wealth of experience and expertise. Discover multiple perspectives on the key issues affecting the CRISPR field by joining roundtable discussions, specifically designed so you can learn from your fellow gene-editing peers.

round tables day 1