13-15th November 2017

Berlin, Germany


Day One
Monday 13th November, 2017

Day Two
Tuesday 14th November, 2017

Networking & Coffee

Chair’s Opening Remarks

Optimising Drug Discovery & Development Through CRISPR

Genome Editing Meets Mouse Modelling for Better Preclinical Tools

  • Danilo Maddalo Lab Head, Oncology Pharmacology, Novartis Institutes for BioMedical Research


  • Genetic manipulations in the mouse
  • How is the CRISPR/Cas9 technology impacting mouse biology
  • Future perspectives for genome editing to generate better preclinical models

CRISPR as a Tool for Target Validation in Drug Discovery


  • CRISPR as a tool in drug discovery to carry out target validation of a list of targets generated in silico and/or by experimental data
  • How to deliver a pipeline to generate KO/KI in primary cells and cell lines through different CRISPR RNP deliveries
  • Routine use of deep sequencing analysis of mutated cells to precisely assess DNA modifications
  • Prospective technology developments using CRISPR to increase the throughput of experiments leading to a faster validation of targets

Developing Genome Wide CRISPR Libraries for Target Discovery

  • Ryan Cawood Chief Executive Officer , Oxford Genetics


  • Developing vector systems for production of large scale DNA libraries
  • High-throughput automation for the CRISPR screening work-flow

Networking Break & Morning Refreshments

Enhancing CRISPR Technology to Drive Innovation in Therapeutic Development

Genome-Scale Synthetic Lethality Screens of Isogenic Cell Lines Reveal Targetable Cancer Vulnerabilities & Functional Gene Networks

  • Rob Wolthuis Head of the Oncogenetics Section, Department of Clinical Genetics, VU Medical Center


  • Investigating the functional characterization of cancer-predisposing genes and the identification of targetable cancer vulnerabilities
  • Discussing a new CCA-wide facility for synthetic lethality-based lentiviral CRISPR screens, as well as custom-directed gene editing protocols to generate precise mutations in human cancer and diploid cell lines
  • Harnessing these approaches to couple with proteomics, mapping of signal transduction, cell biological validations and drug sensitivity analyses
  • Developing genome-wide screening using CRISPR-Cas9 libraries in combination with patient-derived cell models for cancer predispositions and human disease

Synthetic sgRNA Enables Highly Efficient & Consistent CRISPR Editing of Primary Cells for Therapeutic Applications


  • Achieving high editing efficiencies in CRISPR therapeutic applications while maintaining consistency remains a significant challenge
  • Traditional methods for generating guide RNAs can yield molecules of inconsistent length and quality that affect genome editing efficiency
  • We demonstrate that synthetic sgRNA produces consistent editing efficiencies superior to two-piece crRNA:tracrRNA complexes and IVT-derived guides

Antibiotic Resurrection Via Programmable RNA-guided Endonuclease Inactivation of Multiple Resistance Genes


  • Using bacterial cybergenetics to resurrect sensitivity to antibiotics in antimicrobial resistant (AMR) pathogens. Our “Nemesis Symbiotics”, use a programmable RNA­-guided DNA endonuclease gene editing technology  to target beta-lactamase (bla) resistance genes
  • Following delivery on a plasmid vector, a single construct inactivates members of 8 families of bla genes –VIM­, OXA­, NDM­, CTX­M­, KPC­, IMP­, SHV­ and TEM (VONCKIST), so resurrecting sensitivity to beta-lactams
  • For therapeutic applications, Transmids, our novel delivery vectors introduce Symbiotics by infection, when packaged in a ‘phage coat, that can also subsequently spread to other bacteria by plasmid conjugation
  • Multi­functional gene targeting systems may obviate the need for prior diagnostic screens for antibiotic resistance and can be used generally as a companion biological therapeutic together with well­-established antibiotics for both therapeutic treatment of infection as well as by prophylactic treatment preventing the spread of AMR

Networking Lunch

In Vivo Genome Editing: from Proof-of-Concept to Therapeutic Delivery

  • Hao Yin Scientist II, Vertex Pharmaceuticals


  • The principles of biomacromolecule delivery and gene editing
  • Proof-of-concept of in vivo CRISPR
  • Therapeutic delivery of CRISPR
  • Chemical modifications for CRISPR application

Chicken IFITM Knockout Technology for Increased Vaccine Production

  • Mark Fife Group Leader, Genetics & Genomics , The Pirbright Institute


  • Identification of antiviral proteins in the chicken (chIFITM), which have shown that a reduction in chIFITM expression results in an increase in the virus titre in CEFs infected with avian influenza A virus (AIV) H9N2, suggesting that chIFITMs have a functional role in the control of viral infections.
  • It is well established that the rate determining step in the manufacture of numerous vaccines is the induction of antiviral immune responses that prevents the replication of vaccine viruses
  • To generate chIFITM knock-down using cutting edge genetic approaches such the CRISPR/Cas9 system to directly target and knock-out chIFITM expression
  • To observe the effect the knock-down of chIFITM genes expression has on viral titre in avian cell lines (commonly used for vaccine production) infected with Influenza A Virus

Chair’s Closing Remarks

Close of the 2nd CRISPR Europe 2017