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February 23-25 2016 | Boston, USA
•	 Improve Target Specificity & Efficiency of Genome Editing
•	 Enhance Drug Discovery & Screening Applications
•	 Design & Develop Clinical Therapies
2nd
Annual
Rachel Haurwitz 	
Caribou Biosciences
Jon Moore	
Horizon Discovery
Alexandra Glucksmann 	
Editas Medicine
Emmanuelle Charpentier	
Max Planck Institution, Umeå University
George Church 	
Harvard Medical School
Rodger Novak
CRISPR Therapeutics
This is
designed to
describe the
profound
impact CRISPR
is having
on basic
research and
therapeutic
development. It
will explore the
research tools,
bioinformatics
and expertise
needed to
make the
most of this
breakthrough
technology.
Eric Rhodes, Horizon
Lead Partner Additional Event Partners
Tel: +1 212 537 5898 | Email: info@hansonwade.com
RESEARCHED & DEVELOPED BY:crispr-congress.com
Welcome to Those Dedicated to CRISPR
2.Engineer the next generation of humanized
animal models  cell lines for improved
disease modeling and preclinical predictability
2. Harness CRISPR technology for more
accurate gene editing in non-eukaryotic 
bacterial and plant cells
5 Key Benefits to Takeaway
1. Discover innovative ways to measure and
optimize specificity of the CRISPR/Cas9
system in order to reduce off target effects
1. Effectively adopt the lentiviral delivery system
to optimize genome wide targeting from basic
research to drug discovery
3. Develop strategies to perform large scale
genome wide screening from basic research
to disease target identification
3.Utilize CRISPR/Cas9 to develop robust cell-
based cancer immunotherapies including
CAR-Ts and TCRs
4. Learn how to independently design and
optimize CRISPR with novel emerging
enzymes (Cpf1) to enhance the precise
nature of genome editing
4. Understand the regulatory requirements
for CRISPR/Cas9 mediated therapeutic
development as it is poised to demonstrate
clinical utility
5.Enhance the delivery and efficiency of 
CRISPR/Cas9 in primary cells
5.Harness CRISPRi to elevate the regulation of gene
expression through epigenetic modifications
5 Key Ideas to Implement
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Achieve the Full Potential of CRISPR Genome Editing to
Ensure Accuracy  Success
Optimize your CRISPR Design to Efficiently Power Novel Applications in Drug
Discovery, Screening and Therapeutic Development
The 2nd Annual CRISPR Congress will enhance the basic
research, drug discovery and therapeutic applications
of CRISPR technology by overcoming key specificity,
efficiency and delivery challenges needed to improve the
precise editing and repair of the genome.
With the superior applications of CRISPR showing no signs
of relenting, join the leading CRISPR figureheads as they
reveal advanced methodology, strategies and clinical
timelines that maximize the power of precision genome
editing and fulfill its revolutionary potential.
 As well as applying CRISPR to optimize the custom
development of stable cell lines  disease models for
target identification and enhanced drug discovery, CRISPR
Congress 2016 pioneers the translation of CRISPR-based
gene therapies into a clinically relevant transformative
therapy.
As CRISPR continues to create a sea change for genetic
research, not to mention the next class of enzymes on
the horizon, join the CRISPR Congress to deepen your
understanding, minimize off-target effects and elevate
delivery mechanisms for therapeutic use. There is a
a key opportunity for you to network and build future
collaborations with fellow peers to  optimize the application
of CRISPR gene editing technology and transform
your research to the next level.
Why the 2nd CRISPR Congress Should Be in Your Calendar
Speaker Faculty
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
Rachel Haurwitz
President  CEO
Caribou Biosciences
Alexandra Glucksmann
Chief Operating Officer
Editas Medicine
Emmanuelle Charpentier
Professor
Max Planck Institution for
Infection Biology, MIMS
Umeå University
George Church
Professor of Genetics
Harvard Medical School
Jon Moore
CSO
Horizon Discovery
John Feder
Associate Director of
Genome Biology
Bristol-Myers Squibb
Chad Cowan 	
Associate Professor,
Harvard Stem Cell
Institute, Broad Institute
Harvard University
TJ Cradick
Head of Genome Editing
CRISPR Therapeutics
Joel Klappenbach
Director of Applied
Genomics
Merck
Gregory Davis
RD Manager, Molecular
Biotechnology
Sigma-Aldrich
Charles Gersbach
Associate Professor, Director,
Center of Biomolecular 
Tissue Engineering
Duke University   
David Root
Senior Director
Genetic Perturbation
Platform and the Functional
Genomics Consortium
Broad Institute
Channabasavaiah
Gurumurthy
Assistant Professor-
Genetics, Director- Mouse
Genome Engineering Core
Facility, University of
Nebraska Medical Center
Greg Gocal
Senior Vice President
RD
Cibus
Benjamin Kleinstiver
Postdoctoral Fellow,
Keith Joung’s Group,
Massachusetts General
Hospital  Harvard Medical
School  
Emily Leproust 	
CEO 	
Twist Bioscience
Lorenz Mayr
VP  Global Head, Reagents
 Assay Development
AstraZeneca
John Doench
Associate Director,
Genetic Perturbation
Platform, MIT
Harvard University  
Jennifer Berman
Staff Scientist,
Applications, Digital
Biology Center
Bio-Rad Laboratories
Rodger Novak
CEO
CRISPR Therapeutics
Jonathan Gootenberg
Feng Zhang’s Lab, Broad
Institute, MIT
Harvard University  
Jason Potter
Senior Scientist  Manager
Thermo Fisher Scientific
Shawn Zhou
Senior Scientist
Genscript
Wen Xue
Assistant Professor, RNA
Therapeutics Institute,
University of Massachusetts
Anja Smith
Director of RD	
Dharmacon, 	
GE Healthcare
Mark Behlke, 	
Chief Scientific Officer
Integrated DNA
Technologies, Inc
Daniel Anderson
Sam Goldblith Associate
Professor
Massachusetts Institute of
Technology (MIT)
Tom Barnes
CSO
Intellia Therapeutics
Speakers all gave excellent presentations - perfect balance of
viewpoints on leveraging CRISPR technology. I enjoyed the format and
structured networking sessions were well thought out – I appreciated the
opportunity to connect with experts in this emerging field.
Previous CRISPR Congress Attendee Warp Drive Bio LLC
Conference Day One
8.00	 Registration, Breakfast  Networking
9.00	 Chair’s Opening Remarks   
CRISPR the Superior: A Revolution That Speeds On
9.10	 Keynote Interviews: Optimizing CRISPR Design to Efficiently
Power Novel Applications in Drug Discovery, Screening and
Therapeutic Development
The panel will discuss and answer the following questions:
•	How has the novel CRISPR enzyme Cpf1 enhanced the application
CRISPR technology?
•	With continued innovation - Will CRISPR always be at the top or are there
better alternatives emerging to drive gene engineering?
•	How has the industry tried to enhance CRISPR/Cas9 mediated gene-editing in
primary cells?
•	How is the industry addressing the ethical challenges surrounding
genetic editing of germline cells in order to be diligent whilst pushing
scientific boundaries?
•	From a therapeutic perspective, how close are we to demonstrating clinical
utility and what have been the lessons learning in preparing CRISPR-based
gene therapies for human trials?
Emmanuelle Charpentier, Professor,
Max Planck Institution for Infection
Biology, MIMS, 	
Umeå University
Alexandra Glucksmann, Chief
Operating Officer, Editas Medicine
Rodger Novak, CEO,
CRISPR Therapeutics
Rachel Haurwitz, President  CEO,
Caribou Biosciences
Tom Barnes, CSO,
Intellia Therapeutics
10.00	 CRISPR/Cas9 – A Swiss Army Knife for the Identification and
Validation of Novel Drug Targets
•	Learn to accurately analyze data from screens and to functionally
validate them
•	Genome wide screening for gain of function or loss of function screening,
either in exploring the developmental state for basic biology or for screening
for therapeutic targets for diseases (drug development)
•	Discover how to establish CRISPR libraries for target identification
Jon Moore, CSO, Horizon Discovery  
10.30	 Morning Refreshments  Speed Networking
Measuring and Optimizing Specificity of the CRISPR/Cas9 System
11.30	 Keynote Presentation: The CRISPR-Cas9 Revolution in Genome
Engineering: Lessons Learned from Bacteria
•	Addressing the origins of CRISPR-Cas9
•	Discover the mechanisms of CRISPR-Cas9 and to highlight the evolution
of CRISPR-Cas9
Emmanuelle Charpentier, Professor,
Max Planck Institution for Infection
Biology, MIMS, Umeå University
12.15	 Rewriting DNA Synthesis
•	Highlighting the innovative silicon-based platform that is revolutionizing
DNA synthesis
•	Addressing how the platform delivers the power of scale, reduces price
and turn-around time through the miniaturization of reaction volumes
by a factor of 1000
•	Allowing design on a grander scale by simplifying experiments through
circumventing the need to clone; whether it is genes, ready-to-use
oligonucleotide pools or variant libraries
Emily Leproust, CEO, Twist Bioscience
12.25	 Beyond Cas9: Discovering New Tools for Precision Genome
Editing
•	Using smaller, easier to deliver Cas9 orthologs enables new translational
applications for genome editing
•	Cas9 is just one of many programmable nucleases in nature with gene
editing potential
•	The RNA-guided endonuclease Cpf1 expands the genome engineering toolbox
and allows for allele-specific targeting
Jonathan Gootenberg, Feng Zhang’s
Lab, Harvard University
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Tuesday February 23rd 2016
16.55 	 Breakout Roundtables: Advancing CRISPR Technology to The Next Level
Discover multiple perspectives on the key issues and applications in the revolutionary CRISPR field by joining roundtable
discussions, specifically designed so you can learn from your fellow gene-editing peers.
Moderators: 	
Joel Klappenbach, Director of Applied Genomics, Merck
Daniel Anderson, Sam Goldblith Associate Professor, Massachusetts Institute of Technology (MIT)  
Discovering how to
perform high throughout
genome screenings
in the development of
CRISPR libraries
5.
Collaborating to
develop and define
protocols for gene
editing in non-
eukaryotic cells
4.
Advancing non-viral delivery
methods to progress
CRISPR-therapeutics – how
can you refine methodology
to overcome this key hurdle
of CRISPR therapeutic
development in the context
of clinical utility
3.Harnessing CRISPRi
technology to fast-track
development of therapies
– ensuring sequence
specific control of
gene expression
2.Evaluating and combining
different methods to
enhance specificity
of CRISPR to minimize
unwanted
off-target effects
1.
12.55	 Ultra-Sensitive Quantification of Genome Editing Events by
Droplet Digital PCR
•	Genome editing events can be rare and difficult to quantify, with frequencies
5% in some targeted cell types
•	Genome editing methods development and optimization would benefit from 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
Jennifer Berman, Staff Scientist,
Applications, Digital Biology Center,
Bio-Rad Laboratories
13.25	 Networking Lunch
14.25	 Ensuring Successful Translation from the Preclinical to Clinical
Stages Using CRISPR/Cas9
•	Addressing the safety aspects involving specificity
•	Learning from other gene editing tools in the clinic including TALENs and ZFNs
Alexandra Glucksmann, Chief
Operating Officer, Editas Medicine
14.55	 Genome Editing Solutions - New Workflow Tools for Genome
Editing
•	High Efficiency editing in difficult cell lines using cas9 mRNA or RNPS
•	Improved delivery using Neon electroporation or Lipofectamine CRISPRMAX
Jason Potter, Senior Scientist 
Manager,
Thermo Fisher Scientific
15.25	 How Epigenome Editing Can Be Used to Reprogram Cell
Phenotype for Disease Modeling and Regenerative Medicine
•	Insight into how epigenome editing tools have been developed to perturb gene
regulatory elements
•	Annotating the function of regulatory elements can be used to discover new
drug targets, diagnostics, and strategies for personalized medicine
Charles Gersbach,	
Associate Professor, Director, Center of
Biomolecular  Tissue Engineering,
Duke University
15.55	 Afternoon Refreshments  Poster Session
16.25	 Beyond the Break: Options for Donor-DNA Driven Genome Editing
with High Fidelity and Efficiency
•	Donor DNA formats and their impact on DNA repair rates
•	Learn to manipulate DNA repair outcomes among diverse cell types
•	Strategies for pooled screens using CRISPR and donor-DNA libraries
Gregory Davis, RD Manager,
Molecular Biotechnology, Sigma-
Aldrich
17.55 	 Chair’s Closing Remarks Jon Moore, CSO, Horizon Discovery  
18.00 	 Evening Drinks Reception Hosted by Horizon Discovery
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Conference Day Two
8.00	 Breakfast  Networking
9.00	 Chair’s Opening Remarks
9.10 	 Genome Engineering Technologies and Applications
•	Using Cas9 to mutate up to 62 sites in one cell simultaneously
•	Applications include xenotransplantation, and gene drives to
eliminate malaria
George Church, Professor of Genetics, 	
Harvard Medical School
Advancing Large Scale Genome Wide Screening
9.40	 Working Together to Unlock the Potential of Precise
Genome Editing
•	Case studies where CRISPR/Cas has been the key enabler for doing
drug discovery
•	Understanding the evolving role of CRISPR/Cas9 technology for
target discovery, target validation and mechanistic studies
•	Leveraging external academic and industrial research collaborations
for genome-wide target discovery
Lorenz Mayr, VP  Global Head, Reagents 
Assay Development, AstraZeneca
10.10	 Innovative Trait Development Tools in Plant Breeding will be
Crucial for Doubling Global Agricultural Productivity by 2050
•	Rapid Trait Development System (RTDS™) employs Gene Repair
OligoNucleobases (GRONs) to make defined spelling changes in
genomic DNA
•	We report that RTDS can significantly improve the outcome of
double strand break activity by reliably inducing precise and targeted
nucleotide spelling changes closely aligned to the cut site
•	Our work demonstrates the significance of gene editing to rapidly,
precisely and reliably improve cro performance to develop any trait
in commercially relevant crop varieties
Greg Gocal, Senior Vice President, Research 
Development, Cibus
10.40	 Opportunities and Challenges of Applied Genome
Engineering Technologies in the Pharmaceutical  
Research Setting
•	Genome engineering is advancing at an incredible pace however,
applications across the types of translational and predicative cell
models now preferred for drug target discovery remains a challenge
•	What areas of the drug discovery process will genome engineering
have the biggest and most immediate impact
•	How does the pharmaceutical industry evaluate and implement a
technology that is evolving so rapidly
John Feder, Associate Director of Genome
Biology, Bristol-Myers Squibb
11.10	 Morning Refreshments
11.40	 Improving CRISPR-Cas9 Gene Knockout with a Validated
Guide RNA Algorithm
•	Development of an algorithm for improved CRISPR RNA functionality
and specificity
•	Utility of a synthetic two-RNA approach for high-throughput arrayed
gene knockout
•	Importance of rigorous alignment tools for CRISPR RNA specificity
Anja Smith, Director of RD, 	
Dharmacon, GE Healthcare
Wednesday February 24th 2016
Harnessing CRISPR Technology in Disease and Preclinical Modeling
12.10 	 Optimizing Bioinformatic Tools to Improve Guide Design
and to Enhance Specificity
•	Optimize CRISPR/Cas systems to drive gene editing
•	Utilize CRISPR corrects the underlying disease-causing mutations
•	Harness bioinformatics and design strategies to improve and
ensure specificity
•	Discovering the use of new CRISPR systems
TJ Cradick, Head of Genome Editing,
CRISPR Therapeutics
12.40	 Genome Editing: From Modeling Disease to Novel
Therapeutics
•	The use of genome editing to generate isogenic cell lines with and
without disease mutations for rigorous human disease modeling,
either by introducing mutation(s) into wild-type human pluripotent
stem cell (hPSC) lines or by “curing” the mutation(s) in patient-
specific human induced pluripotent stem cells (hiPSCs)
•	Utilizing genome editing tools to rapidly and efficiently generate
knockout or mutant alleles of several genes in human pluripotent
stem cells to differentiate both targeted lines and isogenic control
lines into various cell types relevant to metabolic disorders
•	Learn how to assess which system is more efficient in human
pluripotent stem cells by targeting the same genomic sites in more
than a dozen genes using both TALENs and CRISPRs in the same
hESC lines or hiPSC lines
•	Sequencing genome-edited clones to compare the rates of off-target
effects produced by the TALEN and CRISPR systems
Chad Cowan, Associate Professor, Harvard
Stem Cell Institute, Broad Institute, Harvard
University
13.10	 Applying GenCRISPR Efficiently – Pioneering to Make
Genome-Editing Easy
•	GenCRISPR services cover mammalian and bacterial cell
line services
•	GenCRISPR-related reagents and making research easy through
one-stop service solution
Shawn Zhou, Senior Scientist, GenScript
13.20	 Networking Lunch
14.20	 Tipping the CRISPR NHEJ/HDR Imbalance: Strategies 	
for Efficient Generation of Large-Size-DNA Knock-in 	
Animal Models
•	Latest developments in enhancing insertion of larger DNA
molecules using HDR-dependent and HDR-independent
mechanisms
•	The CRISPR strategies that help rapid development of humanized
animal models	
Channabasavaiah Gurumurthy, 	
Assistant Professor-Genetics, Director- Mouse
Genome Engineering Core Facility,
University of Nebraska Medical Center
14.50	 Precision Cancer Mouse Models Through Genome Editing
with CRISPR-Cas9
•	Address how CRISPR can speed up the generation of precision
cancer models
•	How these models can be used to understand the evolution and
progression of individual tumors
•	Identify new strategies for cancer treatment
Wen Xue, Assistant Professor, RNA
Therapeutics Institute, University of
Massachusetts Medical School
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Maximizing Precision for Novel Applications
15.20 	 Increase Efficiency of Genome Editing Using the Alt-R™
CRISPR-Cas9 System
•	Improving on-target CRISPR-Cas9 genome editing performance
while also reducing cell toxicity and eliminating innate cellular
immune response as compared to in vitro transcribed guide
RNA alternatives
•	A discussion on challenges and potential pitfalls of genome editing
with guidance towards successful CRISPR-Cas9 editing experiments
Mark Behlke, Chief Scientific Officer, 	
Integrated DNA Technologies, Inc
15.50	 Afternoon Refreshments
16.20 	 Application of CRISPR Technologies to Genetic Screens
•	As a powerful tool for large scale screens how do CRISPR libraries
perform in actual screens?
•	What factors are experimentally shown to be important?
•	What are strengths and limitations of CRISPR libraries and screens?
David Root, Senior Director, Broad Institute
Genetic Perturbation Platform and the
Functional Genomics Consortium,
Broad Institute
16.50	 Genetic Screens with CRISPR-Cas9: Optimizing On-Target
Activity and Avoiding Off-Target Effects
•	Considerations for library design to optimize on-target activity
•	Detection and avoidance of potential off-target activity
•	Comparison of CRISPR to RNAi
John Doench, Associate Director, Genetic
Perturbation Platform, MIT, Harvard University  
17.20	 Expanding and Improving the Genome-Wide Specificities of
CRISPR-Cas9 Nucleases
•	The targeting range of Cas9 can be expanded by engineering
variants that can target previously inaccessible sites
•	The genome-wide specificity of Cas9 can be improved with novel
evolved variants
•	GUIDE-seq can be used to define the genome-wide specificities of
Cas9 variants
Benjamin Kleinstiver, Postdoctoral Fellow,
Keith Joung’s Group, Massachusetts General
Hospital  Harvard Medical School
17.50 	 Chair’s Closing Remarks   
17.55 	 Evening Drinks Reception Hosted by Sigma-Aldrich
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Workshop A
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
CRISPR technology has rapidly changed the face of biological research. In particular, precision genome editing has been so
readily adopted it has now become routine for many labs. One key application of CRISPR is in the discovery and functional
understanding of genomics through high-throughput screening. In this interactive workshop, attendees will capitalize lessons
learned in order to:
•	 Develop protocols to carry out large scale genome screens to better understand gene interactions in disease
•	 Learn from a range of applications to optimize the development of CRISPR libraries
•	 Optimize data analysis of genome screens for target identification in drug discovery
CRISPR/Cas9-mediated genome editing has become the preferred tool of choice when it
comes to efficiently and easily manipulating the genome with strong specificity. As such,
a rapidly adopted application has emerged to create more physiologically relevant and
predictable in vivo disease models to better understand the complexity of human disease. In
this interactive workshop, attendees will discover solutions and answers to help them:
•	 Outline the latest in developments in the CRISPR-mediated generation of disease models
•	 Overcome the current limitations of using CRISPR for disease modeling
•	 Harness CRISPR gene editing technology to develop humanized animal models,
particularly for emerging immunotherapies
•	 Address and discuss case studies to standardize protocols to utilize CRISPR for
in vivo models
Performing High Throughout Genome Screenings for Developing CRISPR Libraries
Date: Thursday February 25th 2016  | Time: 9.00am – 12.00pm
Developing More Predictable Disease Models With CRISPR: From
Basic Research to Target Identification and Drug Discovery
Date: Thursday February 25th 2016  | Time: 1.00pm – 4.00pm
TJ Cradick, PhD has studied the different families of nucleases that enable genome editing and gene
therapy, such as for correcting mutations that cause diseases. Recent work has focused on CRISPR/Cas
nucleases (see publication list). Previously we developed and studied TAL Effector Nucleases (TALENs),
and Zinc Finger Nucleases (ZFNs). Many of these studies in academia or industry included measuring
and improving specificity. TJ co-authored manuscripts detailing our bioinformatics web tools: ZFN-Site,
PROGNOS, SAPTA and COSMID (CRISPR off-target). 
Workshop Leader
TJ Cradick, Head of Genome Editing, CRISPR Therapeutics
CB Gurumurthy obtained a faculty position at the University of Nebraska Medical Center (UNMC), Omaha in 2007.
He is currently an assistant professor in Developmental Neuroscience Department and serves as the Director of
UNMC Mouse Genome Engineering Core Facility. He is actively pursuing research in developing newer geneome
editing technologies in addition to developing widely-usable animal models for basic and drug discovery research.
Workshop Leader
Channabasavaiah Gurumurthy, Assistant Professor-Genetics, Director- Mouse
Genome Engineering Core Facility, University of Nebraska Medical Center
Workshop B
CRISPR Partners
Sigma-Aldrich
Sigma-Aldrich is proud to offer its newest line of genome editing tools, Sigma CRISPRs, to the global research
community. Sigma CRISPRs offer rapid, reliable and reproducible results – everything you need for gene editing
experiments. Sigma-Aldrich also offers the CRISPR Core Partnership Program providing scientists and core
facilities with world class service and a diverse portfolio of innovative CRISPR reagents, with the support of an
industry-leading bioinformatics engine. The Cell Design Studio team at Sigma Aldrich, utilizes CRISPR, ZFN,
and shRNA technologies to rapidly and efficiently generate model cell lines to be used in basic research, cell-
based assays, target validation and much more.
www.sigmaaldrich.com
Thermo Fisher Scientific
Thermo Fisher Scientific Inc. (NYSE: TMO) is the world leader in serving science, our mission is to enable
our customers to make the world healthier, cleaner and safer. We help our customers accelerate life
Sciences research, solve complex analytical challenges, improve patient diagnostics and increase laboratory
productivity. Through our four premier brands Thermo Scientific, Life Technologies, Fisher Scientific and Unity
Lab Services we offer an unmatched combination of innovative technologies, purchasing convenience and
comprehensive support.
www.thermoscientific.com
Program Partner
Program Partner
Bio-Rad
Bio-Rad Laboratories, Inc. designs, manufactures, and distributes a broad range of innovative tools and
services to the life science research and clinical diagnostics markets. Founded in 1952, Bio-Rad has a
global team of more than 7,750 employees and serves more than 100,000 research and industry customers
worldwide through the company’s global network of operations. Throughout its existence, Bio-Rad has built
strong customer relationships that advance scientific research and development efforts and support the
introduction of new technology used in the growing fields of genomics, proteomics, drug discovery, food safety,
and medical diagnostics.
www.bio-rad.com   
Program Partner
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Horizon Discovery
Horizon Discovery combines long scientific heritage in translational research with GENESIS™, a
precision gene editing platform incorporating rAAV, CRISPR and ZFN technologies. Horizon supplies
genetically-defined cell lines, gene-editing tools and services, custom cell line generation, molecular
reference standards, and contract research services to approaching 1,000 academic, clinical and
biopharmaceutical organisations.
www.horizondiscovery.com
Lead Partner
CRISPR Partners
Dharmacon – part of GE Healthcare
Dharmacon has revolutionized the field of RNA synthesis with the introduction of 2’-ACE synthesis chemistry
since 1995. As leaders in custom RNA synthesis, Dharmacon was an early participant in the newly discovered
field of RNA interference, and contributed several key scientific findings. Dharmacon RNAi products were some
of the first commercially available. This leadership has continued through technical advances in bioinformatics,
and chemical modifications to improve performance. Today, our areas of research and research tools have
expanded to support all aspects of RNAi interference; siRNA, lentiviral shRNA, tools for microRNA research,
and whole-genome scale libraries for RNAi functional screens of genes, microRNAs and long non-coding RNAs.
Dharmacon also has the largest collection of cDNAs and ORFs commercially available.
In addition, Dharmacon offers a unique set of CRISPR-Cas9 gene editing tools. The Dharmacon CRISPR-Cas9
platform greatly simplifies the workflow of permanently knocking out genes. Our approach includes pre-
designed, ready-to-use DNA and RNA components and enables fast assessment of multiple target sites per
gene for multiple genes. We offer CRISPR Guide RNA, high quality, ready-to-use lentiviral and synthetic reagents
to guide Cas9 cleavage;  Cas9 Nuclease for your cell type to ensure robust Cas9 expression or explore DNA-free
options; CRISPR Controls and Detection Primers essential to assessment of CRISPR-Cas9 genomic editing
experiments; CRISPR-Cas9 Pooled sgRNA or arrayed crRNA for high-throughput gene editing studies.
www.dharmacon.gelifesciences.com
Program Partner
Cibus
Cibus has developed plant and microbial platforms enabling it to become a world leader in precision gene
editing, generally, and advanced non-transgenic breeding, specifically. The Rapid Trait Development System
(RTDS™), its proprietary technology for non-transgenic breeding, enables site-specific edits of native genes with
no introduction of foreign DNA. The precise and predictable outcomes can replicate products developed using
traditional mutagenesis and are identical to those that could occur in nature, given enough time. Consequently,
these plants are not considered transgenic, the key attributes of GMO crops.
www.cibus.com
Program Partner
Integrated DNA Technologies
Integrated DNA Technologies (IDT) is a leader in the manufacture and development of products for the
research and diagnostic life science market. The largest supplier of custom nucleic acids in the world, IDT
serves academic research, biotechnology, and pharmaceutical development.
IDT products support a wide variety of applications, including next generation sequencing (NGS), genome
editing, DNA amplification, SNP detection, microarray analysis, expression profiling, gene quantification, and
synthetic biology. Platform-independent NGS products and services are available in addition to DNA and RNA
oligonucleotides, qPCR assays, siRNA duplexes, reagents for CRISPR-Cas9 genome editing, and custom gene
synthesis services.
IDT has an active research and development division that supports product development, and participates in
corporate and academic collaborations. IDT also supports the education of the next generation of scientists
through our regular sponsorship of the international Genetically Engineered Machine (iGEM) competition, and
other scientific outreach activities.
www.idtdna.com
Program Partner
Twist Bioscience
Our expertise is synthetic DNA. We have developed a manufacturing process featuring a 10,000-well silicon
platform capable of producing synthetic biology tools. Our platform overcomes the current inefficiencies
of synthetic DNA production, and enables cost-effective, rapid and high-quality synthetic gene production.
The Twist Bioscience platform has the potential to accelerate the development of personalized medicine,
sustainable chemical production, improved agriculture production as well as new applications such as in vivo
diagnostics, biodetection and data storage.
www.twistbioscience.com     
Spotlight Partner
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Chardan Capital Markets
Chardan Capital Markets, LLC is a privately held investment banking firm with a focus on micro, small, and
mid-cap markets within the healthcare and technology sectors. Our full range of services includes capital
raising, merger and acquisition advisory, strategic advisory, equity research, institutional trading, and market
making. Having established a successful record of completing complex and challenging deals, we’ve become a
leading authority on capital solutions for emerging growth companies.
www.chardancm.com     
Hosting Lunch Partner
ERS Genomics
ERS Genomics was formed to commercialize the foundational CRISPR-Cas9 intellectual property held by
Dr. Emmanuelle Charpentier. Licenses are available for research and sale of products and services across
multiple fields including: research tools, kits, reagents; discovery of novel targets for therapeutic intervention;
cell lines for discovery and screening of novel drug candidates; GMP production of healthcare products;
production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology.
www.ersgenomics.com     
Networking Partner
CRISPR Partners
Advanced Analytical Technologies
Advanced Analytical Technologies, Inc. (AATI), founded in 1998 and located in Ankeny, Iowa, is a world leader in
multi-channel (parallel) capillary electrophoresis. The company’s products are designed to improve processes
within the life science, agricultural, molecular diagnostics, biofuels, biotechnology, and pharmaceutical
industries. Its award winning instrument, the Fragment Analyzer™ is the premier instrument to automate the
analysis of nucleic acid fragments and smears. Designed to improve laboratory workflow and decrease time
to results, the Fragment Analyzer can dependably and reliably assess gene editing events generated through
CRISPR/Cas9 mutagenesis using a streamlined heteroduplexing and cleavage process for ultra-sensitive
detection of fragments and digital data outputs with specially designed software to aid in analysis. Additionally,
Reagent Kits are available to accurately qualify and quantify nucleic acid raw materials like genomic DNA and
RNA and analyze NGS library preparation. The Fragment Analyzer is the premier instrument to automate
analysis of nucleic acids.
www.aati-us.com
Exhibitor
MTI-GlobalStem
MTI-GlobalStem develops high-quality, standardized stem cell reagents and innovative technologies for life
science and neurobiology research. Our products include primary and iPSC-derived neural cells, neuronal
culture media and supplements, pluripotent stem cells and stem cell culture media, feeder cells, and
transfection reagents for hard-to-transfect cells. Our latest transfection products are optimized for large
plasmid delivery of CRISPR/Cas9 vectors into a range of cell types, including hard-to-transfect primary cells.
www.mti-globalstem.com
Exhibitor
GenScript
GenScript is the leading gene, peptide, protein and antibody research partner for fundamental life science
research, translational biomedical research, and early stage pharmaceutical development. Since our
establishment in 2002, GenScript has exponentially grown to become a global leading Contract Research
Organization that provides services and products to scientists in 86 countries worldwide
www.genscript.com     
Spotlight Partner
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
Code:5792
* All discount offers (including team discounts) require payment at the time of registration to receive any discount. ‘Early Bird’ discounts require payment at time of registration and on or before the
cut-off date to receive any discount. All discount offers cannot be combined with any other offer. The conference fee includes lunch, refreshments and course documentation. The fee does not include
travel or hotel accommodation.
Full payment is due on registration. Cancellation and Substitution Policy: Cancellations must be received in writing. If the cancellation is received more than 14 days before the conference attendees
will receive a full credit to a future conference. Cancellations received 14 days or less (including the fourteenth day) prior to the conference will be liable for the full fee. A substitution from the same
organisation can be made at any time.
Changes to Conference  Agenda: Hanson Wade reserves the right to postpone or cancel an event, to change the location or alter the advertised speakers. Hanson Wade is not responsible for any loss
or damage or costs incurred as a result of substitution, alteration, postponement or cancellation of an event for any reason and including causes beyond its control including without limitation, acts of
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Data Protection: The personal information shown and/or provided by you will be held in a database. It may be used to keep you up to date with developments in your industry. Sometimes your details
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Register
Event Prices
Pricing  Venue	
Hyatt Regency Boston	
One Avenue de Lafayette	
Boston, Massachusetts, 	
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Accommodation: Overnight accommodation
is not included in the registration fee,
however accommodation options will
be sent out with your confirmation email
upon registering.
Academic  Not-for-Profit Pricing
Package
Register  	
Pay before Friday 	
January 22nd 2016
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Gold Package: Conference + 2 Workshops
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$1898
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Workshop Each: $499
Industry Pricing
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Register  	
Pay before Friday January 	
22nd 2016
Standard Prices
Gold Package: Conference + 2 Workshops
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$3797
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$3098
(save $300)
$3198
(save $200)
Bronze Package:	
Conference Only
$2499
(save $200)
$2699
Workshop Each: $699
Tel: +1 212 537 5898      Email: info@hansonwade.com
crispr-congress.com     #CRISPR2016     CRISPR Precision Genome Editing
2nd
Annual CRISPR Precision Gene Editing Congress
Boston, USA | February 23-25 2016
• 10% discount – 3 delegates
• 15% discount – 4 delegates
• 20% discount – 5 or more delegates
Please note that discounts are only valid when three or more
delegates from one company book and pay at the same time.
Team Discounts*
crispr-congress.com/register	
Tel: +1 212 537 5898
Email: register@hansonwade.com
Mail: Hanson Wade
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London, SW1W 0AU

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2nd CRISPR Congress Boston, 23-25 February 2016

  • 1. February 23-25 2016 | Boston, USA • Improve Target Specificity & Efficiency of Genome Editing • Enhance Drug Discovery & Screening Applications • Design & Develop Clinical Therapies 2nd Annual Rachel Haurwitz Caribou Biosciences Jon Moore Horizon Discovery Alexandra Glucksmann Editas Medicine Emmanuelle Charpentier Max Planck Institution, Umeå University George Church Harvard Medical School Rodger Novak CRISPR Therapeutics This is designed to describe the profound impact CRISPR is having on basic research and therapeutic development. It will explore the research tools, bioinformatics and expertise needed to make the most of this breakthrough technology. Eric Rhodes, Horizon Lead Partner Additional Event Partners Tel: +1 212 537 5898 | Email: info@hansonwade.com RESEARCHED & DEVELOPED BY:crispr-congress.com
  • 2. Welcome to Those Dedicated to CRISPR 2.Engineer the next generation of humanized animal models cell lines for improved disease modeling and preclinical predictability 2. Harness CRISPR technology for more accurate gene editing in non-eukaryotic  bacterial and plant cells 5 Key Benefits to Takeaway 1. Discover innovative ways to measure and optimize specificity of the CRISPR/Cas9 system in order to reduce off target effects 1. Effectively adopt the lentiviral delivery system to optimize genome wide targeting from basic research to drug discovery 3. Develop strategies to perform large scale genome wide screening from basic research to disease target identification 3.Utilize CRISPR/Cas9 to develop robust cell- based cancer immunotherapies including CAR-Ts and TCRs 4. Learn how to independently design and optimize CRISPR with novel emerging enzymes (Cpf1) to enhance the precise nature of genome editing 4. Understand the regulatory requirements for CRISPR/Cas9 mediated therapeutic development as it is poised to demonstrate clinical utility 5.Enhance the delivery and efficiency of  CRISPR/Cas9 in primary cells 5.Harness CRISPRi to elevate the regulation of gene expression through epigenetic modifications 5 Key Ideas to Implement Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016 Achieve the Full Potential of CRISPR Genome Editing to Ensure Accuracy Success Optimize your CRISPR Design to Efficiently Power Novel Applications in Drug Discovery, Screening and Therapeutic Development The 2nd Annual CRISPR Congress will enhance the basic research, drug discovery and therapeutic applications of CRISPR technology by overcoming key specificity, efficiency and delivery challenges needed to improve the precise editing and repair of the genome. With the superior applications of CRISPR showing no signs of relenting, join the leading CRISPR figureheads as they reveal advanced methodology, strategies and clinical timelines that maximize the power of precision genome editing and fulfill its revolutionary potential.  As well as applying CRISPR to optimize the custom development of stable cell lines disease models for target identification and enhanced drug discovery, CRISPR Congress 2016 pioneers the translation of CRISPR-based gene therapies into a clinically relevant transformative therapy. As CRISPR continues to create a sea change for genetic research, not to mention the next class of enzymes on the horizon, join the CRISPR Congress to deepen your understanding, minimize off-target effects and elevate delivery mechanisms for therapeutic use. There is a a key opportunity for you to network and build future collaborations with fellow peers to  optimize the application of CRISPR gene editing technology and transform your research to the next level. Why the 2nd CRISPR Congress Should Be in Your Calendar
  • 3. Speaker Faculty 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016 Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing Rachel Haurwitz President CEO Caribou Biosciences Alexandra Glucksmann Chief Operating Officer Editas Medicine Emmanuelle Charpentier Professor Max Planck Institution for Infection Biology, MIMS Umeå University George Church Professor of Genetics Harvard Medical School Jon Moore CSO Horizon Discovery John Feder Associate Director of Genome Biology Bristol-Myers Squibb Chad Cowan Associate Professor, Harvard Stem Cell Institute, Broad Institute Harvard University TJ Cradick Head of Genome Editing CRISPR Therapeutics Joel Klappenbach Director of Applied Genomics Merck Gregory Davis RD Manager, Molecular Biotechnology Sigma-Aldrich Charles Gersbach Associate Professor, Director, Center of Biomolecular Tissue Engineering Duke University David Root Senior Director Genetic Perturbation Platform and the Functional Genomics Consortium Broad Institute Channabasavaiah Gurumurthy Assistant Professor- Genetics, Director- Mouse Genome Engineering Core Facility, University of Nebraska Medical Center Greg Gocal Senior Vice President RD Cibus Benjamin Kleinstiver Postdoctoral Fellow, Keith Joung’s Group, Massachusetts General Hospital Harvard Medical School   Emily Leproust CEO Twist Bioscience Lorenz Mayr VP Global Head, Reagents Assay Development AstraZeneca John Doench Associate Director, Genetic Perturbation Platform, MIT Harvard University   Jennifer Berman Staff Scientist, Applications, Digital Biology Center Bio-Rad Laboratories Rodger Novak CEO CRISPR Therapeutics Jonathan Gootenberg Feng Zhang’s Lab, Broad Institute, MIT Harvard University   Jason Potter Senior Scientist Manager Thermo Fisher Scientific Shawn Zhou Senior Scientist Genscript Wen Xue Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Anja Smith Director of RD Dharmacon, GE Healthcare Mark Behlke, Chief Scientific Officer Integrated DNA Technologies, Inc Daniel Anderson Sam Goldblith Associate Professor Massachusetts Institute of Technology (MIT) Tom Barnes CSO Intellia Therapeutics Speakers all gave excellent presentations - perfect balance of viewpoints on leveraging CRISPR technology. I enjoyed the format and structured networking sessions were well thought out – I appreciated the opportunity to connect with experts in this emerging field. Previous CRISPR Congress Attendee Warp Drive Bio LLC
  • 4. Conference Day One 8.00 Registration, Breakfast Networking 9.00 Chair’s Opening Remarks CRISPR the Superior: A Revolution That Speeds On 9.10 Keynote Interviews: Optimizing CRISPR Design to Efficiently Power Novel Applications in Drug Discovery, Screening and Therapeutic Development The panel will discuss and answer the following questions: • How has the novel CRISPR enzyme Cpf1 enhanced the application CRISPR technology? • With continued innovation - Will CRISPR always be at the top or are there better alternatives emerging to drive gene engineering? • How has the industry tried to enhance CRISPR/Cas9 mediated gene-editing in primary cells? • How is the industry addressing the ethical challenges surrounding genetic editing of germline cells in order to be diligent whilst pushing scientific boundaries? • From a therapeutic perspective, how close are we to demonstrating clinical utility and what have been the lessons learning in preparing CRISPR-based gene therapies for human trials? Emmanuelle Charpentier, Professor, Max Planck Institution for Infection Biology, MIMS, Umeå University Alexandra Glucksmann, Chief Operating Officer, Editas Medicine Rodger Novak, CEO, CRISPR Therapeutics Rachel Haurwitz, President CEO, Caribou Biosciences Tom Barnes, CSO, Intellia Therapeutics 10.00 CRISPR/Cas9 – A Swiss Army Knife for the Identification and Validation of Novel Drug Targets • Learn to accurately analyze data from screens and to functionally validate them • Genome wide screening for gain of function or loss of function screening, either in exploring the developmental state for basic biology or for screening for therapeutic targets for diseases (drug development) • Discover how to establish CRISPR libraries for target identification Jon Moore, CSO, Horizon Discovery 10.30 Morning Refreshments Speed Networking Measuring and Optimizing Specificity of the CRISPR/Cas9 System 11.30 Keynote Presentation: The CRISPR-Cas9 Revolution in Genome Engineering: Lessons Learned from Bacteria • Addressing the origins of CRISPR-Cas9 • Discover the mechanisms of CRISPR-Cas9 and to highlight the evolution of CRISPR-Cas9 Emmanuelle Charpentier, Professor, Max Planck Institution for Infection Biology, MIMS, Umeå University 12.15 Rewriting DNA Synthesis • Highlighting the innovative silicon-based platform that is revolutionizing DNA synthesis • Addressing how the platform delivers the power of scale, reduces price and turn-around time through the miniaturization of reaction volumes by a factor of 1000 • Allowing design on a grander scale by simplifying experiments through circumventing the need to clone; whether it is genes, ready-to-use oligonucleotide pools or variant libraries Emily Leproust, CEO, Twist Bioscience 12.25 Beyond Cas9: Discovering New Tools for Precision Genome Editing • Using smaller, easier to deliver Cas9 orthologs enables new translational applications for genome editing • Cas9 is just one of many programmable nucleases in nature with gene editing potential • The RNA-guided endonuclease Cpf1 expands the genome engineering toolbox and allows for allele-specific targeting Jonathan Gootenberg, Feng Zhang’s Lab, Harvard University Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016 Tuesday February 23rd 2016
  • 5. 16.55 Breakout Roundtables: Advancing CRISPR Technology to The Next Level Discover multiple perspectives on the key issues and applications in the revolutionary CRISPR field by joining roundtable discussions, specifically designed so you can learn from your fellow gene-editing peers. Moderators: Joel Klappenbach, Director of Applied Genomics, Merck Daniel Anderson, Sam Goldblith Associate Professor, Massachusetts Institute of Technology (MIT) Discovering how to perform high throughout genome screenings in the development of CRISPR libraries 5. Collaborating to develop and define protocols for gene editing in non- eukaryotic cells 4. Advancing non-viral delivery methods to progress CRISPR-therapeutics – how can you refine methodology to overcome this key hurdle of CRISPR therapeutic development in the context of clinical utility 3.Harnessing CRISPRi technology to fast-track development of therapies – ensuring sequence specific control of gene expression 2.Evaluating and combining different methods to enhance specificity of CRISPR to minimize unwanted off-target effects 1. 12.55 Ultra-Sensitive Quantification of Genome Editing Events by Droplet Digital PCR • Genome editing events can be rare and difficult to quantify, with frequencies 5% in some targeted cell types • Genome editing methods development and optimization would benefit from 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 Jennifer Berman, Staff Scientist, Applications, Digital Biology Center, Bio-Rad Laboratories 13.25 Networking Lunch 14.25 Ensuring Successful Translation from the Preclinical to Clinical Stages Using CRISPR/Cas9 • Addressing the safety aspects involving specificity • Learning from other gene editing tools in the clinic including TALENs and ZFNs Alexandra Glucksmann, Chief Operating Officer, Editas Medicine 14.55 Genome Editing Solutions - New Workflow Tools for Genome Editing • High Efficiency editing in difficult cell lines using cas9 mRNA or RNPS • Improved delivery using Neon electroporation or Lipofectamine CRISPRMAX Jason Potter, Senior Scientist Manager, Thermo Fisher Scientific 15.25 How Epigenome Editing Can Be Used to Reprogram Cell Phenotype for Disease Modeling and Regenerative Medicine • Insight into how epigenome editing tools have been developed to perturb gene regulatory elements • Annotating the function of regulatory elements can be used to discover new drug targets, diagnostics, and strategies for personalized medicine Charles Gersbach, Associate Professor, Director, Center of Biomolecular Tissue Engineering, Duke University 15.55 Afternoon Refreshments Poster Session 16.25 Beyond the Break: Options for Donor-DNA Driven Genome Editing with High Fidelity and Efficiency • Donor DNA formats and their impact on DNA repair rates • Learn to manipulate DNA repair outcomes among diverse cell types • Strategies for pooled screens using CRISPR and donor-DNA libraries Gregory Davis, RD Manager, Molecular Biotechnology, Sigma- Aldrich 17.55 Chair’s Closing Remarks Jon Moore, CSO, Horizon Discovery 18.00 Evening Drinks Reception Hosted by Horizon Discovery Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016
  • 6. Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016 Conference Day Two 8.00 Breakfast Networking 9.00 Chair’s Opening Remarks 9.10 Genome Engineering Technologies and Applications • Using Cas9 to mutate up to 62 sites in one cell simultaneously • Applications include xenotransplantation, and gene drives to eliminate malaria George Church, Professor of Genetics, Harvard Medical School Advancing Large Scale Genome Wide Screening 9.40 Working Together to Unlock the Potential of Precise Genome Editing • Case studies where CRISPR/Cas has been the key enabler for doing drug discovery • Understanding the evolving role of CRISPR/Cas9 technology for target discovery, target validation and mechanistic studies • Leveraging external academic and industrial research collaborations for genome-wide target discovery Lorenz Mayr, VP Global Head, Reagents Assay Development, AstraZeneca 10.10 Innovative Trait Development Tools in Plant Breeding will be Crucial for Doubling Global Agricultural Productivity by 2050 • Rapid Trait Development System (RTDS™) employs Gene Repair OligoNucleobases (GRONs) to make defined spelling changes in genomic DNA • We report that RTDS can significantly improve the outcome of double strand break activity by reliably inducing precise and targeted nucleotide spelling changes closely aligned to the cut site • Our work demonstrates the significance of gene editing to rapidly, precisely and reliably improve cro performance to develop any trait in commercially relevant crop varieties Greg Gocal, Senior Vice President, Research Development, Cibus 10.40 Opportunities and Challenges of Applied Genome Engineering Technologies in the Pharmaceutical Research Setting • Genome engineering is advancing at an incredible pace however, applications across the types of translational and predicative cell models now preferred for drug target discovery remains a challenge • What areas of the drug discovery process will genome engineering have the biggest and most immediate impact • How does the pharmaceutical industry evaluate and implement a technology that is evolving so rapidly John Feder, Associate Director of Genome Biology, Bristol-Myers Squibb 11.10 Morning Refreshments 11.40 Improving CRISPR-Cas9 Gene Knockout with a Validated Guide RNA Algorithm • Development of an algorithm for improved CRISPR RNA functionality and specificity • Utility of a synthetic two-RNA approach for high-throughput arrayed gene knockout • Importance of rigorous alignment tools for CRISPR RNA specificity Anja Smith, Director of RD, Dharmacon, GE Healthcare Wednesday February 24th 2016
  • 7. Harnessing CRISPR Technology in Disease and Preclinical Modeling 12.10 Optimizing Bioinformatic Tools to Improve Guide Design and to Enhance Specificity • Optimize CRISPR/Cas systems to drive gene editing • Utilize CRISPR corrects the underlying disease-causing mutations • Harness bioinformatics and design strategies to improve and ensure specificity • Discovering the use of new CRISPR systems TJ Cradick, Head of Genome Editing, CRISPR Therapeutics 12.40 Genome Editing: From Modeling Disease to Novel Therapeutics • The use of genome editing to generate isogenic cell lines with and without disease mutations for rigorous human disease modeling, either by introducing mutation(s) into wild-type human pluripotent stem cell (hPSC) lines or by “curing” the mutation(s) in patient- specific human induced pluripotent stem cells (hiPSCs) • Utilizing genome editing tools to rapidly and efficiently generate knockout or mutant alleles of several genes in human pluripotent stem cells to differentiate both targeted lines and isogenic control lines into various cell types relevant to metabolic disorders • Learn how to assess which system is more efficient in human pluripotent stem cells by targeting the same genomic sites in more than a dozen genes using both TALENs and CRISPRs in the same hESC lines or hiPSC lines • Sequencing genome-edited clones to compare the rates of off-target effects produced by the TALEN and CRISPR systems Chad Cowan, Associate Professor, Harvard Stem Cell Institute, Broad Institute, Harvard University 13.10 Applying GenCRISPR Efficiently – Pioneering to Make Genome-Editing Easy • GenCRISPR services cover mammalian and bacterial cell line services • GenCRISPR-related reagents and making research easy through one-stop service solution Shawn Zhou, Senior Scientist, GenScript 13.20 Networking Lunch 14.20 Tipping the CRISPR NHEJ/HDR Imbalance: Strategies for Efficient Generation of Large-Size-DNA Knock-in Animal Models • Latest developments in enhancing insertion of larger DNA molecules using HDR-dependent and HDR-independent mechanisms • The CRISPR strategies that help rapid development of humanized animal models Channabasavaiah Gurumurthy, Assistant Professor-Genetics, Director- Mouse Genome Engineering Core Facility, University of Nebraska Medical Center 14.50 Precision Cancer Mouse Models Through Genome Editing with CRISPR-Cas9 • Address how CRISPR can speed up the generation of precision cancer models • How these models can be used to understand the evolution and progression of individual tumors • Identify new strategies for cancer treatment Wen Xue, Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Medical School Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016
  • 8. Maximizing Precision for Novel Applications 15.20 Increase Efficiency of Genome Editing Using the Alt-R™ CRISPR-Cas9 System • Improving on-target CRISPR-Cas9 genome editing performance while also reducing cell toxicity and eliminating innate cellular immune response as compared to in vitro transcribed guide RNA alternatives • A discussion on challenges and potential pitfalls of genome editing with guidance towards successful CRISPR-Cas9 editing experiments Mark Behlke, Chief Scientific Officer, Integrated DNA Technologies, Inc 15.50 Afternoon Refreshments 16.20 Application of CRISPR Technologies to Genetic Screens • As a powerful tool for large scale screens how do CRISPR libraries perform in actual screens? • What factors are experimentally shown to be important? • What are strengths and limitations of CRISPR libraries and screens? David Root, Senior Director, Broad Institute Genetic Perturbation Platform and the Functional Genomics Consortium, Broad Institute 16.50 Genetic Screens with CRISPR-Cas9: Optimizing On-Target Activity and Avoiding Off-Target Effects • Considerations for library design to optimize on-target activity • Detection and avoidance of potential off-target activity • Comparison of CRISPR to RNAi John Doench, Associate Director, Genetic Perturbation Platform, MIT, Harvard University   17.20 Expanding and Improving the Genome-Wide Specificities of CRISPR-Cas9 Nucleases • The targeting range of Cas9 can be expanded by engineering variants that can target previously inaccessible sites • The genome-wide specificity of Cas9 can be improved with novel evolved variants • GUIDE-seq can be used to define the genome-wide specificities of Cas9 variants Benjamin Kleinstiver, Postdoctoral Fellow, Keith Joung’s Group, Massachusetts General Hospital Harvard Medical School 17.50 Chair’s Closing Remarks 17.55 Evening Drinks Reception Hosted by Sigma-Aldrich Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016
  • 9. Workshop A Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016 CRISPR technology has rapidly changed the face of biological research. In particular, precision genome editing has been so readily adopted it has now become routine for many labs. One key application of CRISPR is in the discovery and functional understanding of genomics through high-throughput screening. In this interactive workshop, attendees will capitalize lessons learned in order to: • Develop protocols to carry out large scale genome screens to better understand gene interactions in disease • Learn from a range of applications to optimize the development of CRISPR libraries • Optimize data analysis of genome screens for target identification in drug discovery CRISPR/Cas9-mediated genome editing has become the preferred tool of choice when it comes to efficiently and easily manipulating the genome with strong specificity. As such, a rapidly adopted application has emerged to create more physiologically relevant and predictable in vivo disease models to better understand the complexity of human disease. In this interactive workshop, attendees will discover solutions and answers to help them: • Outline the latest in developments in the CRISPR-mediated generation of disease models • Overcome the current limitations of using CRISPR for disease modeling • Harness CRISPR gene editing technology to develop humanized animal models, particularly for emerging immunotherapies • Address and discuss case studies to standardize protocols to utilize CRISPR for in vivo models Performing High Throughout Genome Screenings for Developing CRISPR Libraries Date: Thursday February 25th 2016 | Time: 9.00am – 12.00pm Developing More Predictable Disease Models With CRISPR: From Basic Research to Target Identification and Drug Discovery Date: Thursday February 25th 2016 | Time: 1.00pm – 4.00pm TJ Cradick, PhD has studied the different families of nucleases that enable genome editing and gene therapy, such as for correcting mutations that cause diseases. Recent work has focused on CRISPR/Cas nucleases (see publication list). Previously we developed and studied TAL Effector Nucleases (TALENs), and Zinc Finger Nucleases (ZFNs). Many of these studies in academia or industry included measuring and improving specificity. TJ co-authored manuscripts detailing our bioinformatics web tools: ZFN-Site, PROGNOS, SAPTA and COSMID (CRISPR off-target).  Workshop Leader TJ Cradick, Head of Genome Editing, CRISPR Therapeutics CB Gurumurthy obtained a faculty position at the University of Nebraska Medical Center (UNMC), Omaha in 2007. He is currently an assistant professor in Developmental Neuroscience Department and serves as the Director of UNMC Mouse Genome Engineering Core Facility. He is actively pursuing research in developing newer geneome editing technologies in addition to developing widely-usable animal models for basic and drug discovery research. Workshop Leader Channabasavaiah Gurumurthy, Assistant Professor-Genetics, Director- Mouse Genome Engineering Core Facility, University of Nebraska Medical Center Workshop B
  • 10. CRISPR Partners Sigma-Aldrich Sigma-Aldrich is proud to offer its newest line of genome editing tools, Sigma CRISPRs, to the global research community. Sigma CRISPRs offer rapid, reliable and reproducible results – everything you need for gene editing experiments. Sigma-Aldrich also offers the CRISPR Core Partnership Program providing scientists and core facilities with world class service and a diverse portfolio of innovative CRISPR reagents, with the support of an industry-leading bioinformatics engine. The Cell Design Studio team at Sigma Aldrich, utilizes CRISPR, ZFN, and shRNA technologies to rapidly and efficiently generate model cell lines to be used in basic research, cell- based assays, target validation and much more. www.sigmaaldrich.com Thermo Fisher Scientific Thermo Fisher Scientific Inc. (NYSE: TMO) is the world leader in serving science, our mission is to enable our customers to make the world healthier, cleaner and safer. We help our customers accelerate life Sciences research, solve complex analytical challenges, improve patient diagnostics and increase laboratory productivity. Through our four premier brands Thermo Scientific, Life Technologies, Fisher Scientific and Unity Lab Services we offer an unmatched combination of innovative technologies, purchasing convenience and comprehensive support. www.thermoscientific.com Program Partner Program Partner Bio-Rad Bio-Rad Laboratories, Inc. designs, manufactures, and distributes a broad range of innovative tools and services to the life science research and clinical diagnostics markets. Founded in 1952, Bio-Rad has a global team of more than 7,750 employees and serves more than 100,000 research and industry customers worldwide through the company’s global network of operations. Throughout its existence, Bio-Rad has built strong customer relationships that advance scientific research and development efforts and support the introduction of new technology used in the growing fields of genomics, proteomics, drug discovery, food safety, and medical diagnostics. www.bio-rad.com Program Partner Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016 Horizon Discovery Horizon Discovery combines long scientific heritage in translational research with GENESIS™, a precision gene editing platform incorporating rAAV, CRISPR and ZFN technologies. Horizon supplies genetically-defined cell lines, gene-editing tools and services, custom cell line generation, molecular reference standards, and contract research services to approaching 1,000 academic, clinical and biopharmaceutical organisations. www.horizondiscovery.com Lead Partner
  • 11. CRISPR Partners Dharmacon – part of GE Healthcare Dharmacon has revolutionized the field of RNA synthesis with the introduction of 2’-ACE synthesis chemistry since 1995. As leaders in custom RNA synthesis, Dharmacon was an early participant in the newly discovered field of RNA interference, and contributed several key scientific findings. Dharmacon RNAi products were some of the first commercially available. This leadership has continued through technical advances in bioinformatics, and chemical modifications to improve performance. Today, our areas of research and research tools have expanded to support all aspects of RNAi interference; siRNA, lentiviral shRNA, tools for microRNA research, and whole-genome scale libraries for RNAi functional screens of genes, microRNAs and long non-coding RNAs. Dharmacon also has the largest collection of cDNAs and ORFs commercially available. In addition, Dharmacon offers a unique set of CRISPR-Cas9 gene editing tools. The Dharmacon CRISPR-Cas9 platform greatly simplifies the workflow of permanently knocking out genes. Our approach includes pre- designed, ready-to-use DNA and RNA components and enables fast assessment of multiple target sites per gene for multiple genes. We offer CRISPR Guide RNA, high quality, ready-to-use lentiviral and synthetic reagents to guide Cas9 cleavage;  Cas9 Nuclease for your cell type to ensure robust Cas9 expression or explore DNA-free options; CRISPR Controls and Detection Primers essential to assessment of CRISPR-Cas9 genomic editing experiments; CRISPR-Cas9 Pooled sgRNA or arrayed crRNA for high-throughput gene editing studies. www.dharmacon.gelifesciences.com Program Partner Cibus Cibus has developed plant and microbial platforms enabling it to become a world leader in precision gene editing, generally, and advanced non-transgenic breeding, specifically. The Rapid Trait Development System (RTDS™), its proprietary technology for non-transgenic breeding, enables site-specific edits of native genes with no introduction of foreign DNA. The precise and predictable outcomes can replicate products developed using traditional mutagenesis and are identical to those that could occur in nature, given enough time. Consequently, these plants are not considered transgenic, the key attributes of GMO crops. www.cibus.com Program Partner Integrated DNA Technologies Integrated DNA Technologies (IDT) is a leader in the manufacture and development of products for the research and diagnostic life science market. The largest supplier of custom nucleic acids in the world, IDT serves academic research, biotechnology, and pharmaceutical development. IDT products support a wide variety of applications, including next generation sequencing (NGS), genome editing, DNA amplification, SNP detection, microarray analysis, expression profiling, gene quantification, and synthetic biology. Platform-independent NGS products and services are available in addition to DNA and RNA oligonucleotides, qPCR assays, siRNA duplexes, reagents for CRISPR-Cas9 genome editing, and custom gene synthesis services. IDT has an active research and development division that supports product development, and participates in corporate and academic collaborations. IDT also supports the education of the next generation of scientists through our regular sponsorship of the international Genetically Engineered Machine (iGEM) competition, and other scientific outreach activities. www.idtdna.com Program Partner Twist Bioscience Our expertise is synthetic DNA. We have developed a manufacturing process featuring a 10,000-well silicon platform capable of producing synthetic biology tools. Our platform overcomes the current inefficiencies of synthetic DNA production, and enables cost-effective, rapid and high-quality synthetic gene production. The Twist Bioscience platform has the potential to accelerate the development of personalized medicine, sustainable chemical production, improved agriculture production as well as new applications such as in vivo diagnostics, biodetection and data storage. www.twistbioscience.com Spotlight Partner Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016
  • 12. Chardan Capital Markets Chardan Capital Markets, LLC is a privately held investment banking firm with a focus on micro, small, and mid-cap markets within the healthcare and technology sectors. Our full range of services includes capital raising, merger and acquisition advisory, strategic advisory, equity research, institutional trading, and market making. Having established a successful record of completing complex and challenging deals, we’ve become a leading authority on capital solutions for emerging growth companies. www.chardancm.com Hosting Lunch Partner ERS Genomics ERS Genomics was formed to commercialize the foundational CRISPR-Cas9 intellectual property held by Dr. Emmanuelle Charpentier. Licenses are available for research and sale of products and services across multiple fields including: research tools, kits, reagents; discovery of novel targets for therapeutic intervention; cell lines for discovery and screening of novel drug candidates; GMP production of healthcare products; production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology. www.ersgenomics.com Networking Partner CRISPR Partners Advanced Analytical Technologies Advanced Analytical Technologies, Inc. (AATI), founded in 1998 and located in Ankeny, Iowa, is a world leader in multi-channel (parallel) capillary electrophoresis. The company’s products are designed to improve processes within the life science, agricultural, molecular diagnostics, biofuels, biotechnology, and pharmaceutical industries. Its award winning instrument, the Fragment Analyzer™ is the premier instrument to automate the analysis of nucleic acid fragments and smears. Designed to improve laboratory workflow and decrease time to results, the Fragment Analyzer can dependably and reliably assess gene editing events generated through CRISPR/Cas9 mutagenesis using a streamlined heteroduplexing and cleavage process for ultra-sensitive detection of fragments and digital data outputs with specially designed software to aid in analysis. Additionally, Reagent Kits are available to accurately qualify and quantify nucleic acid raw materials like genomic DNA and RNA and analyze NGS library preparation. The Fragment Analyzer is the premier instrument to automate analysis of nucleic acids. www.aati-us.com Exhibitor MTI-GlobalStem MTI-GlobalStem develops high-quality, standardized stem cell reagents and innovative technologies for life science and neurobiology research. Our products include primary and iPSC-derived neural cells, neuronal culture media and supplements, pluripotent stem cells and stem cell culture media, feeder cells, and transfection reagents for hard-to-transfect cells. Our latest transfection products are optimized for large plasmid delivery of CRISPR/Cas9 vectors into a range of cell types, including hard-to-transfect primary cells. www.mti-globalstem.com Exhibitor GenScript GenScript is the leading gene, peptide, protein and antibody research partner for fundamental life science research, translational biomedical research, and early stage pharmaceutical development. Since our establishment in 2002, GenScript has exponentially grown to become a global leading Contract Research Organization that provides services and products to scientists in 86 countries worldwide www.genscript.com Spotlight Partner Tel: +1 212 537 5898 Email: info@hansonwade.com crispr-congress.com #CRISPR2016 CRISPR Precision Genome Editing 2nd Annual CRISPR Precision Gene Editing Congress Boston, USA | February 23-25 2016
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