February 21-23 2017

Boston, USA


Day One
Tuesday February 21st, 2017

Day Two
Wednesday February 22nd, 2017

08.00
Registration & Coffee

09.00
Chair’s Opening Remarks

  • Terence Flotte Dean of the School of Medicine, Professor of Medical Education , University of Massachusetts Medical School

09.05
Panel Discussion: Bringing CRISPR into the Clinic: Making CRISPR Therapeutics a Reality

Synopsis

The panel will discuss and answer the following questions:

  • What are the main disease areas in which CRISPR derived therapeutics are being developed?
  • What organs/tissues are showing the most promise in terms of CRISPR complex delivery?
  • How does the CRISPR regulatory landscape compare to other gene editing tools?
  • What is the timeline for CRISPR ex vivo therapeutics entering Phase I trials?
  • What’s on the horizon for future CRISPR therapeutic applications?

10.00
Rewriting DNA Synthesis

Synopsis

Think Big, Screen Once: Genome Scale Editing with Custom CRISPR Libraries

  • To provide an overview on CRISPR library design philosophy as well as discuss the impact of high quality DNA synthesis on enabling accurate gene editing
  • The intelligent CRISPR library design algorithms from Desktop Genetics
  • Twist Bioscience’s 9,600-well silicon-based DNA synthesis platform
  • How high quality oligo pools from Twist Bioscience generate more accurate and uniform sgRNA libraries
  • Specific examples of how CRISPR libraries are used in research pipelines, accelerating the development of novel molecular therapeutics

10.30
Morning Refreshments & Speed Networking

Chaired by Terence Flotte, Dean of the School of Medicine, Professor of Medical Education, University of Massachusetts Medical School

11.30 Defining and Improving the Genome-Wide Specificities of CRISPR-Cas Nucleases

  • Genome editing targeting range can be expanded by engineering variants that can target previously inaccessible sites
  • The genome-wide specificities of Cas nucleases can be improved with novel evolved variants
  • Methods to define the genome-wide specificities of Cas nucleases

Benjamin Kleinstiver, Instructor, Keith Joung’s Group, Massachusetts General Hospital & Harvard Medical School

12.00 CIRCLE-Seq, a Highly Sensitive In Vitro Genome-Wide Method for Defining CRISPR-Cas9 Nuclease Off-Targets

  • CIRCLE-seq is a novel, highly sensitive, in vitro method to enrich and sensitively detect unintended CRISPR-Cas9 “off-target” cleavage in bulk human genomic DNA
  • In contrast to existing in vitro methods, CIRCLE-seq selectively sequences Cas9-cleaved DNA fragments
  • CIRCLE-seq detects all or nearly all sites previously detected by GUIDE-seq as well as many additional bona fide low-frequency sites

Shengdar Tsai, Assistant Member, Department of Hematology, St. Jude Children’s Research Hospital

12.30 A Systematic CRISPR/Cas9-Mediated Approach to Create Endogenously Tagged Human iPSC Lines

  • A 3 part CRISPR/Cas9 system to introduce GFP into various genomic loci in human iPS cells
  • A gene editing workflow that includes screens and QC assays to ensure precise editing while retaining stem cell characteristics
  • Creation of a collection of clonal gene edited lines that help understand stem cell organization and activities such as cell division and differentiation
  • These gene edited cells will be used to better understand cell behavior in both normal and pathological contexts with live imaging

Ru Gunawardane, Director of Stem Cells & Gene editing, Allen Institute for Cell Science

13.00 Networking Lunch

14.00 Genome Editing Using AsCpf1 Protein and Chemically Modified crRNAs as an RNP

  • AsCpf1 recognizes a ‘TTTN’ PAM site which enables genome editing in AT-rich sequences
  • AsCpf2 can be used with electroporation of a ribonuclear protein complex (RNP) comprising the recombinant protein with a synthetic crRNA
  • Peak efficiency of Cpf1 RNP electroporation requires use of an electroporation enhancer
  • Chemically-modified variants of the Cpf1 crRNA that improve genome editing using this AsCpf1 RNP

Mark Behlke, Chief Scientific Officer, Integrated DNA Technologies

14.30 Detecting In Vivo Off-Targets for Cas9 and Cpf1

  • Developing a protocol for accurate off-target detection
  • Comparing off-target profiles of Cas9 vs. Cpf1 in model cell lines

Winston Yan, Graduate Student, Feng Zhang’s Lab, McGovern Broad MIT

15.00 CRISPR-Cas9 Screening with Arrayed Synthetic crRNA Libraries

  • Discuss key factors for success with arrayed crRNA screening
  • Discover benefits of synthetic dual CRISPR guide RNA
  • Evaluate screening results using high content analysis with a cell cycle reporter line

Anja Smith, Director of R&D, Dharmacon (part of GE Healthcare)

11.30 Transforming and Translating Drug Discovery – Use of the CRISPR/Cas9 Technology in Target Discovery, Hit Finding and Translational Studies

  • Overview of PGE and introduction to CRISPR/Cas9 technology
  • Applications in target finding, hit and lead discovery, efficacy and safety models
  • AstraZeneca’s unique approach towards innovation and collaboration
  • Case studies from AstraZeneca’s drug discovery programs
  • Future outlook of PGE for drug discovery and therapeutic applications

Lorenz Mayr, VP & Global Head, Reagents & Assay Development, AstraZeneca

12.00 Knockout Screening with the Sanger Whole Genome Arrayed CRISPR Libraries

  • The discovery and adaptation of the CRISPR pathway has made the prospect of high-throughput knockout screening a reality
  • To introduce the world’s first arrayed whole genome CRISPR screening libraries, produced through a collaboration between MilliporeSigma and the Welcome Trust Sanger Institute
  • Discuss the promise that these new tools hold for uncovering novel drug and disease targets

Shawn Shafer, Director of Advanced Genomics, MilliporeSigma

12.15 Streamlining CRISPR-Mediated Transcriptome Modulation

  • Catalytically-dead Cas9 has shown great promise as a tool for directing suppression or activation of endogenous loci
  • Current approaches for CRISPR-mediated gene activation rely on co-expression of separate transcription modifiers for maximal effect
  • Describe systems for simplified, robust, multicomponent CRISPR-based gene activation

Benjamin Haley, Senior Scientist, Department of Molecular Biology, Genentech

12.45 iTOP: A Novel Non-Viral Delivery System for Gene- Editing Based Therapeutic

  • Development of the iTOP technology, a proprietary platform technology for the intracellular delivery of bioactive molecules
  • Highly efficient in vivo delivery of recombinant proteins
  • In vivo application in CRISPR-Cas9 mediated gene repair

Niels Geijsen, Founder & Scientific Advisor, NTrans, Technologies BV

13.00 Networking Lunch

14.00 CRISPR Functional Genomic Screening For Drug Discovery and Translational Research

  • Impact and application of CRISPR functional genomic technologies in drug discovery research environment
  • Case examples of CRISPR screening for new target discovery
  • Areas of unmet needs in CRISPR technologies

Namjin Chung, Head of Functional Genomics, Target Enabling Science & Technology, Discovery Research, AbbVie

14.30 Arrayed CRISPR in High Throughput Screening – A Viable Alternative to Pooled Based Approaches?

  • Highlighting the pros and cons of pooled based and wellbased CRISPR
  • Introduce the different guide modalities and compare feasibility
  • Discuss initial results of pilot studies

Marc Hild, Senior Investigator I, Novartis Institutes for BioMedical Research

15.00 Therapeutic Advances Using CRISPR

  • Extensive studies with S. pyogenes robustly show high activity
  • Careful choice of gRNAs results in high specificity
  • Recent advances result in high levels of homology directed repair (HDR)

TJ Cradick, Head of Gene Editing, CRISPR Therapeutics

15.30
Afternoon Refreshments & Poster Session sponsored by Twist Bioscience

Synopsis

With an agenda packed with case studies and data-driven presentations, the CRISPR Boston poster session will allow you deeper access into the most innovative research. Meet the scientists conducting exciting research and pick their brains to gain insights into the emerging CRISPR gene editing applications. Apply their learnings straight into your own work, cultivate research collaborations and leave inspired by new ideas.

16.15
Breakout Roundtables: Advancing CRISPR Technology to The Next Level

Synopsis

Drive your own learning and crowd source ideas. The CRISPR speaker faculty is second to none but there is just as much knowledge in the audience as there is onstage. Tap into this wealth of experience and expertise to 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.

  • Evaluating and combining different bioinformatics methods to enhance specificity
  • Harnessing CRISPRi technology to fasttrack development of therapies
  • Advancing non-viral delivery methods to progress CRISPRtherapeutics
  • Optimizing protocols for gene editing in primary cells
  • Utilizing non-traditional organism disease models

17.15
Chair’s Closing Remarks

  • Terence Flotte Dean of the School of Medicine, Professor of Medical Education , University of Massachusetts Medical School

17.20
Drinks Reception Hosted by Twist Bioscience