2nd CRISPR Precision Genome Editing congress Berlin 2017 Agenda

Maximise the Impact of
Precision Gene Editing to
Drive Biomedical Applications
& Future Therapeutic
Breakthroughs
Tel: +44 (0)203 141 8700 Email: info@hansonwade.com CRISPR Precision Gene Editing
Expert speakers include:
CRISPR Europe 2017 Partners:
Kristine Freude
Associate Professor
University of
Copenhagen
Conrad Lichtenstein
Chief Scientific Officer
Nemesis Bioscience
Lin Wu
Director, Genome
Modification Facility
Harvard University
Matteo Martufi
Senior Scientist
GSK
Dan Blat
Senior Scientist- Manager
Immunocore
David Parry-Smith
Senior Scientific Manager
Wellcome Trust Sanger
Institute
13-15 November 2017 Berlin, Germany
www.crispr-europe.com
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REGISTER TO ATTEND

The CRISPR meeting was a
great central hotspot, meeting
those at the heart of this
exciting technology
CRISPR Europe Attendee, Ntrans
Technologies BV
I greatly enjoyed the diversity
and topic coverage of the talks,
thanks for putting together an
excellent program!
CRISPR Boston Attendee, Syros
Pharmaceuticals
Good conference with a lot
of relevant speakers
CRISPR Europe Attendee, GSK
2nd CRISPR Precision Genome Editing Congress Europe
13-15 November 2017 | Berlin, Germany
Tel: +44 (0)20 3141 8700 Email: info@hansonwade.com
www.crispr-europe.com CRISPR Precision Gene Editing
2
Optimise CRISPR
in your workflows
Uncover advanced
bioinformatics
tools for off-
target detection
Advance precision
gene editing in
disease models
Incorporate
precision base
editing
Implement
novel delivery
methodologies
Improve the
application of
CRISPR in iPS cells
Enhance precision
editing in primary
cells
Discoverthe
impactofCRISPR
inantiviraldrug
development
Increase in vivo
knock in efficiency
UncoverCRISPR-
mediatedTCR
therapeutic
advancements
1.
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6.
2.
7.
3.
8.
4.
9.
5.
10.
Hearwhat previous
attendees have to say
from CRISPR EU:
Uncoverthe latest advancements of
CRISPR gene editing transforming
therapeutic R&D
Discover howyour peers are accelerating precision genome
editing to the next level to realise the full potential of this
unstoppable technology
With CRISPR officially cemented as the editing tool transforming basic research
and pharma R&D, the 2nd CRISPR Precision Genome Editing Europe 2017 is
the forum to keep up to date with vital developments in a industry showing no
signs of slowing down.
CRISPR Europe Congress is back with the key technology and industry
developments that are uncovering more and more innovative applications of
this breakthrough technology. From basic research to therapeutic development,
this end to end CRISPR congress will optimise your CRISPR workflow. Experience
the full value of this technology in base editing, primary cell editing, invivo
delivery, antiviral drugs & gene therapies.
With recent data questioning ‘precision’ editing, now is the time to ensure your
CRISPR studies are fully optimised for greater specificity & efficiency with
advanced verification protocols and off-target analysis. Join your peers and
discover how they are developing innovative solutions to advance delivery
methodologies for biomedical research to advance future breakthrough
therapeutic applications.
With the adoption and advancement ofCRISPR technology rapidly progressing,
make sure you attend CRISPR Europe 2017 to stay ahead ofthis fast-paced industry.
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Advance your understanding of CRISPR
technology & uncover novel applications

Expert Speakers
Kristine Freude
Associate
Professor, Group
of Stem Cells
& Embryology,
Department of
Veterinary &
Animal Sciences
University of
Copenhagen
Lin Wu
Director, Genome
Harvard University
Louise Baskin
Senior Product
Manger
Dharmacon- a
GE Healthcare
Company
Conrad
Lichtenstein
Chief Scientific
Officer
Nemesis Bioscience
Danilo Maddalo
Laboratory Head
Novartis
Matteo Martufi
Senior Scientist
GSK
Mark Fife
Group Leader,
Genetics &
Genomics
The Pirbright
Institute
Viresh Patel
Global Marketing
Director,
Digital Biology
Group
Bio-Rad
Laboratories
Dan Blat
Senior Scientist-
Manager
Immunocore
Eric Paul
Bennett
Associate
Professor
University of
Copenhagen
David Parry-Smith
Group Leader, Stem
Cell Informatics
Wellcome Trust
Sanger Institute
Ryan Cawood
Chief Executive
Officer
Oxford Genetics
Pedro Costa
Sir Henry Wellcome
Postdoctoral Fellow
King’s College
London
Kevin Holden
Head of Synthetic
Biology
Synthego
Hao Yin
Postdoctoral Fellow
MIT
It was a great opportunity to meet people working on similar projects
and to discuss issues with them. It was also a great environment to network
with people from both industry and academia
CRISPR Europe Attendee, Bio-Rad
3

Conference DayOne | Monday 13th November 2017
8.00 Registration & Coffee
9.00 Chair’s Opening Remarks
Applications of Precision Gene Editing Technology in Biomedical Research
Eric Paul Bennett
Associate Professor
University of
Copenhagen
9.10 Cellular Indel Profiles & Dynamics Induced by Different CRISPR/Cas9
Delivery Formats: Applying Ex Vivo Methodologies In Vivo
• Plasmid-, piggyback-, lenti- and RNP -Cas9 and gRNA delivery methodologies
• Cas9 indel formation detection methodologies: Pro’s and con’s of the available
methods
• Indel formation dynamics and profiles induced byvarious CRISPR/Cas9 delivery
methods
Viresh Patel
Global Marketing
Director,
Digital Biology Group
Bio-Rad
Laboratories
9.40 Sensitive Quantification of Genome Editing Events by Droplet
Digital PCR
• Development and optimisation of genome editing methods for a sensitive, rapid-
readout tool for edit validation and off-target detection
• Droplet Digital PCR (ddPCR) enables sensitive (<0.1%), precise absolute
quantification of NHEJ and HDR alleles in a rapid, high-throughput format
10.10 Speed Networking & Morning Refreshments
David Parry-Smith
Group Leader, Stem
Cell Informatics
Wellcome Trust
Sanger Institute
11.10 Learning from CRISPR/Cas-9: Advancing Bioinformatics for Precision
Gene Editing
• An informatics perspective on genome editing with CRISPR/Cas9
• Improving on-target and off-target computational analysis for designing genome
editing experiments
• Examples of feedback from high-throughput experiments to guide learning from
data and development of novel algorithms
Louise Baskin
Senior Product
Manger
Dharmacon- a GE
Healthcare Company
11.40 Hit Identification & Confirmation Strategies in Arrayed CRISPR-Cas9
Screening
• Execution of a synthetic crRNA library screen using high content imaging identify
potential hits important in the cell cycle
• Statistical examination of six phenotypic parameters reveal robust, functional
knockout for multiple reagents per gene
• Follow-up experiments including confirmation of phenotype in an independent
experiment, expression analysis, and editing efficiency will be discussed
• Gene annotation and crRNA target position analysis give novel insights into system
biology
• Confirmation of hits with orthogonal reagents (siRNA) supports high-confidence
hits
• Strategies for further validation and hit stratification will be discussed
4

12.10 Networking Lunch
Development of Advanced Disease Models Through Precision Gene Editing
Lin Wu
Director, Genome
Harvard University
13.10 CRISPR/Cas9 Genome Editing in Mice
• Based on analysis of the current data, the efficiency for gene KO through NHEJ has
been significantly higher (~50%) than gene KI through HDR or HR (~15%)
• Reagents of different forms of Cas9 and gRNA used for gene editing
• Targeted gene disruption and deletion to generate gene KO mice
• Site-specific transgene integration to generate gene KI mice
Kristine Freude
Associate Professor,
Group of Stem Cells
& Embryology,
Department of
Veterinary & Animal
Sciences
University of
Copenhagen
13.40 Modelling of Neurodegenerative Diseases in a Dish with the Help of
CRISPR-Cas9
• Challenges and possibilities forCRISPR-Cas9 gene editing in cellularmodels of
neurodegeneration
• Gene specific hurdles forCRISP-Cas9 gene editing
• Example ofa disease model forfrontotemporal dementia linked to chromosome 3
Dan Blat
Senior Scientist-
Manager, Pre-
clinical Biology
Immunocore Ltd
14.10 Employing the CRISPR/Cas9 System in Pre-Clinical Testing of T cell
Receptor-Based Bispecific Drug Candidates
• Overviewofthe ImmTAC platform:TCR-based bispecific biologics forcancer
immunotherapy
• The unique requisiteswhen deleting/editing HLA-presented peptides in cell lines
• Ourcurrentyet everchanging CRISPRworkflow
• Discussion ofsome ofthe technical details and observationswe have made throughout our
CRISPRwork
14.40 Afternoon Refreshments & Poster Session
Pedro Costa
Sir Henry Wellcome
Postdoctoral Fellow
King’s College London
15.20 Advancing CRISPR Technology to The Next Level
Driveyourown learning and crowd source ideas.The CRISPR speakerfacultyis second to none but there isjust as much
knowledge in the audience as there is onstage.Tap into thiswealth ofexperience and expertise to discovermultiple
perspectives on the keyissues affecting the CRISPR field byjoining roundtable discussions, specificallydesigned soyou can
learn fromyourfellowgene-editing peers.
Harnessing CRISPR
to understand
disease
mechanisms in in
vivo models
Modulating
epigenetic
factors using
CRISPR gene
editing
HarnessingCRISPRi
orCRISPRafor
interferenceor
augmentationof
geneexpressionasa
drugdiscoverytool
Developing
human iPS cells
for therapeutic
development
of monoclonal
antibodies
1. 2. 3. 4.
16.20 Close of Day 1
5

Conference DayTwo | Tuesday 14th November 2017
8.00 Networking & Coffee
9.00 Chair’s Opening Remarks
Optimising Drug Discovery & Development Through CRISPR Technology
Danilo Maddalo
Laboratory Head
Novartis
9.10 Genome Editing Meets Mouse Modelling for Better Preclinical Tools
• Genetic manipulations in the mouse
• How is the CRISPR/Cas9 technology impacting mouse biology
• Future perspectives for genome editing to generate better preclinical models
Matteo Martufi
Senior Scientist
GSK
9.40 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
Ryan Cawood
Chief Executive
Officer
Oxford Genetics
10.10 Developing Genome Wide CRISPR Libraries for Target Discovery
• Developing vector systems for production of large scale DNA libraries
• High-throughput automation for the CRISPR screening work-flow
10.40 Networking Break & Morning Refreshments
Enhancing CRISPR Technology to Drive Innovation in Therapeutic Development
Kevin Holden
Head of Synthetic
Biology
Synthego
11.10 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
Conrad Lichtenstein
Chief Scientific
Officer
Nemesis Bioscience
11.40 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
6

12.10 Networking Lunch
Hao Yin
Postdoctoral Fellow
MIT
13.40 In Vivo Delivery of CRISPR/Cas9 Genome Editing
• What technology developments are leading the industry one step closer in
delivering CRISPR/Cas9 in vivo?
• Advancing non-viral delivery for gene-editing to specific tissues
• What is on the horizon to enhance in vivo delivery methodologies?
Mark Fife
Group Leader,
Genetics & Genomics
The Pirbright
Institute
14.10 Chicken IFITM Knockout Technology for Increased Vaccine Production
• 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
14.40 Chair’s Closing Remarks
14.45 Close of the 2nd CRISPR Europe 2017
Part of the CRISPR Event Series
7

Workshop Day | Wednesday 15th November 2017
Inthisinteractiveworkshop,youwilldiscover
solutionsandanswerstohelpyou:
• Advance CRISPR genome editing in transgenic mice
• Overcome the current limitations ofusing CRISPR forinsertions for
higherefficiencyofdesired edits
• Uncoverprotocol advancements foradvanced invivo editingwith
optimised specificity
• Learn about the different applications ofCRISPR-edited transgenic
mice in a biomedical research setting
Thisworkshopwilladdressanddiscussthefollowing
topics:
• What technologydevelopments are leading industryone step closer
in delivering CRISPR/Cas9 invivo?
• Which tissues/organs are showing the most promise forCRISPR in
vivo delivery?
• Outlining novel non-viral deliverymethodologies forfuture gene
therapies
• Highlighting future developments in invivo CRISPR complex delivery
Optimising the Development & Application of CRISPR
Generated Transgenic Mice Models
Optimising In Vivo Delivery for The Advancement of CRISPR
Therapeutics
Workshop Leader:
Lin Wu, Director,
Genome Modification
Facility, Harvard
University
Workshop Leader:
Hao Yin
Postdoctoral Fellow
MIT
WorkshopA: 9:00 - 11:30
Workshop B: 12:00 - 14:30
Afterworking manyyears as an
associate directorat theTransgenic
and GeneTargeting Facilityof
Massachusetts General Hospital,
Harvard Medical School, Lin
became the directorofthe Genome
Modification Facility(GMF) at Harvard
Universityin 2012.The GMFprovides
transgenic, gene targeting, and other
services to investigators ofHarvard
Universityand its affiliated institutions,
aswell as to investigatorswithin the
US and abroad.
It was a great
opportunity to learn and
network
CRISPR Boston Attendee,
Texas A&M University
8

2017 COMMERCIAL PARTNERS
ProgrammePartner
Bio-Rad Laboratories, Inc. designs,
manufactures, and distributes a
broad range ofinnovative tools
and services to the life science
research and clinical diagnostics markets. Founded in 1952,
Bio-Rad has a global team ofmore than 7,750 employees
and serves more than 100,000 research and industry
customersworldwide through the company’s global network
ofoperations.Throughout its existence, Bio-Rad has built
strong customerrelationships that advance scientific research
and development efforts and support the introduction of
newtechnologyused in the growing fields ofgenomics,
proteomics, drug discovery, food safety, and medical
diagnostics.
www.bio-rad.com
ProgrammePartner
Dharmacon offers a unique set of
CRISPRCas9 gene editing tools. Our
approach includes pre-designed,
ready-to-use DNAand RNAcomponents and enables fast
assessment ofmultiple target sites pergene formultiple
genes.We offerCRISPR Guide RNA, high quality, ready-to-use
lentiviral and synthetic reagents to guide Cas9 cleavage.
www.dharmacon.gelifesciences.com
SpotlightPartner
Synthego is a leading providerofgenome
engineering solutions.The company’s
flagship product, CRISPRevolution, is
a portfolio ofsynthetic RNAdesigned forCRISPR genome
editing and research. Synthego’svision is to bring precision
and automation to genome engineering, enabling rapid
and cost-effective researchwith consistent results forevery
scientist. Headquartered in SiliconValley, California, Synthego
customers include leading institutions in 31 countries around
theworld, and 9 ofthe top 10 global biologyuniversities.
www.synthego.com
Exhibitor
Biolegiowas founded in 1996 in Nijmegen,
the Netherlands.The first production
facilitywas located on the campus ofthe
Radboud Universityin Nijmegen.Afterseveralyears ofgrowth
the companyneeded more space to keep upwith demands
and moved to a newbuilding.This newbuilding, still located
in Nijmegen, offered the spacewe needed forexpanding our
endeavors.The product range has expanded from custom
made oligonucleotides to highlymodified oligonucleotides,
fittedwith everycommerciallyavailable modification. Making
them suitable forawide range ofapplications including (Next
Generation) Sequencing, PCR, Real-Time PCR, SNPdetection,
genotyping, gene expression and mutation detection.
www.biolegio.com
SpotlightPartner
Oxford Genetics is a synthetic biology
companytaking an engineering
approach to DNA,virus and cell line
design, optimization and development.The business has been
developing solutions that improve all aspects ofmammalian
gene expression, therebymaximizing the productivityof
protein, antibodyandvirus systems. Ourresearch has involved
the testing ofawide arrayofsequence types, including
proprietarypromoters and untranslated regions, and the
extensive screening ofexogenous proteins that influence cell
biologyand system output.This screening has been coupled
with in house algorithms forthe batch optimization ofgenes
and high-throughput design forDNAassembly.These skill sets
have allowed us build a track record ofdelivering DNAdesign
and cell line development projects forthe improved production
ofantibodies,vaccines andviruses.
www.oxfordgenetics.com
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Diane McKenna
Commercial Director
T. +44 (0)20 3141 8700
E. sponsor@hansonwade.com
Partnerwithus
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2nd CRISPR Precision Genome Editing congress Berlin 2017 Agenda