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

Colliding Cell Therapy with Precision CRISPR

Using CRISPR/Cas9 to Illuminate Stem Cell Organization and Dynamics


  • A systematic approach for generating knock-ins in stem cells
  • Identifying and improving precision of HDR events
  • Use of live imaging and gene editing to explore cell states

Gene Delivery & Gene Editing in the Retina


  • Overview of methods for “in vivo” delivery to retina
  • Genes delivery to different cell types in retina
  • Crispr/Cas9 mediated gene editing in the retina

Using CRISPR to Modulate Immunosuppressive Pathways in CAR T Cell Products


  • Identified a highly specific gRNA for knockout of the TGFβRII gene in primary CAR T cells using CRISPR
  • Validated that TGFβRII KO mediated CAR T cell products resistant to TGFβ-mediated suppression
  • Identified a TGFβ RNA signature that is induced when CAR T cells see antigen expressing tumor cells in the presence of TGFβ; this signature is reversed when TGFβRII is knocked out by CRISPR

Morning Refreshments

11.10 Nucleic Acid Detection with RNA-Guided RNA Targeting CRISPR Cas13

  • Discovery of novel CRISPR enzymes, including CRISPR-Cas13
  • Biochemical characterization of CRISPR-Cas13 for RNA targeting
  • Development of CRISPR-Cas13 for highly sensitive and specific nucleic acid diagnostics

Jonathan S. Gootenberg, Ph.D. Candidate, Broad Institute of MIT and Harvard

11.40 Enhanced CRISPR/Cas9-Mediated Precise Genome Editing by Improved Design and Delivery of gRNA, Cas9 Nuclease & Donor DNA

  • Efficient knock-in strategies for SNPs, small insertions and epitope tags
  • Design of donors for best integration
  • Improved cas9 protein

Jason Potter, Senior Scientist, Thermo Fisher

12.10 In Vivo CRISPR Models in the Drug Discovery Process

  • Impact of genome editing in DD
  • How CRISPR models compare with previous models
  • Future perspectives and applications

Danilo Madalo, Laboratory Head, Novartis

12.40 Enhanced Proofreading Governs CRISPR-Cas9 Targeting Accuracy

  • DNA target binding reorients Cas9 protein domains to trigger nuclease activity
  • A region of the protein called REC3 functions as a master controller for protein activation
  • Minor alterations within the REC3 domain alters the differential between on- and off-target activity and results in a hyper-accurate Cas9 protein (HypaCas9)

Janice Chen, Scientist – Jennifer Doudna’s lab, Berkeley

11.10 Large Scale Production of Engineered T Cells Using CRISPR/Cas9

  • Gee editing enhances potential of CAR-T cells therapy
  • Sourcing of cGMP grade guide RNA, Cas9 and RNPs
  • Understanding of gene editing in manufacturing setting

Shantanu Kumar, Senior Scientist, Juno Therapeutics

11.40 High Fidelity Genome Editing Using RNP Complexes with a Novel Mutant HiFi Cas9

  • Off-target effects (OTEs) remain a problem for genome editing, even using RNP methods
  • Several mutant Cas9 enzymes have been described that lower OTEs but have low on-target activity when used in RNP methods
  • A new HiFi Cas9 was developed with high on-target and low off-target activity as RNP

Mark A. Behlke, Chief Scientific Officer, Integrated DNA Technologies

12.10 CRISPR Editing in Hematopoietic Stem and Progenitor Cells for the Treatment of Hematologic Diseases

  • Optimisation of delivery methods
  • Sustainable editing over long term (Engraftment of human CD34+ cells in NSG mice)
  • Genotype : Phenotype relationship – determining the desirable edits

Jack Heath, Senior Research Associate, Editas Medicine

12.40 CAMT: Genome Editing Rescue of a Novel MPL Mutant in Primary Cord Blood HSPCs

  • Function of mutant Mpl receptor can be rescued using 2 approaches: autophagy-dependent cell surface delivery and CRISPR-Cas9 gene Editing
  • Edited primary cord blood CD34+ retained their ability to differentiate into platelet-producing megakaryocytes in vitro
  • HDR and NHEJ editing events, alone or in combination, were able to restore the receptor’ function
  • Proof-of-principle for a direct therapeutic application of genome editing in CAMT

Cedric Cleyrat, Assistant Research Professor & Genome Editing Consultant, UNM Cancer Centre

Networking Lunch

14.00 CRISPR-Cas9 Screening to Identify Cancer Dependencies

  • Use of high-throughput genome-scale CRISPR-Cas9 screening to identify cancer dependencies
  • Identification and reconciliation of copy number effects in CRISPR-Cas9 negative selection screens
  • Future directions for CRISPR-Cas9 technology in cancer biology

Andrew Aguirre, Medical Oncologist, Dana-Farber Cancer Institute

14.30 CRISPR Genetic Screens: An Effective Tool to Identify Genes Responsible for Disease Pathologies and Drug Efficacy

  • Complex pools of lentiviral-based sgRNA libraries provide one of the most effective tools to uncover the functional genetic elements that regulate disease pathologies, drug efficacy, and other biological responses.
  • Modifications of the standard sgRNA structure improve  effectiveness and CRISPR library performance.
  • Results of CRISPR-based gene inhibition and activation (CRISPRi and CRISPRa) screens differ considerably depending on the sgRNA design of the libraries

Paul Diehl, COO, Cellecta

14.45 Harnessing RNA targeting CRISPR systems for transcriptome engineering and human health

  • Background on key CRISPR-Cas13 RNA targeting features
  • Development of CRISPR-Cas13 for mammalian RNA knockdown and RNA viral therapeutics
  • Application of CRISPR-Cas13 for RNA enrichment and imaging
  • Application of CRISPR-Cas13 for therapeutic RNA editing

Omar O. Abudayyeh, Scientist, MIT

15.15 CRISPR Functional Screening for Human Primary Cells

  • Utilizing FACS sorting as a readout

Chen-Lin Hsieh, Senior Scientist, Abbvie

14.00 Using CRISPR to Generate Potent Allogeneic CAR T Therapeutics

  • CRISPR/cas9 enables multiplex editing in T Cells
  • Recombinant allogeneic CAR T cells are readily manufactured
  • System is readily applicable to many cancer targets

Jon Terret, Head of Immuno Oncology, CRISPR Therapeutics

14.30 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

Kevin Holden, Head of Synthetic Biology, Synthego Corporations

14.45 Application of CRISPR Method to Interpret GWAS in COPD

  • Connecting non-coding variants in GWAS loci with disease susceptible genes in COPD
  • Phenotypic recapitulation of human COPD in murine genetic models
  • Application of CRISPR/Cas-9 targeting COPD GWAS locus

Anny Xiaobo Zhou, Assistant Professor, Harvard

15.00 CRISPR Genetic Circuits for Controllable Gene Therapies

  • Safety and controllability are two important features of future CRISPR therapies
  • Synthetic biology can enable control over the CRISPR technology for more sophisticated gene therapies
  • Synthetic CRISPR circuits and similar strategies can lead us towards this direction

Samira Kiani, Assistant Professor, Arizona State Univeristy

Afternoon refreshments

Gene Editing By CRISPR-Cas & Cpf1 In The Mouse Brain


  • AAV-based delivery strategies for gene editing in the brain
  • Single and multiplex editing using CRISPR-Cas and Cpf1 in neurons
  • Nuclei sorting of brain cells for analyzing genomic DNA and RNA

Safety Package and the Methods for Progression to CRISPR Clinical Trials


  • Effective on-target editing
  • Lack of detectible off-target events using genome-wide and bioinformatics-derived assays
  • Additional testing for safety package

Chair’s Closing Remarks

Close of the 4th Annual Precision CRSIPR Congress 2018