Personalising CRISPR Genome Editing || Edward Perello || Disruptor Stories

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PERSONALISING CRISPRGENOME EDITINGAugust 2016Edward Perello| CBO & Founder

Desktop Genetics Ltd. 2016 An Illumina-backed company

@EdwardPerello | @DesktopGenetics

PRIVILEGED AND CONFIDENTIAL

Riley Doyle () - Insert GUIDE PICKER GIFDESKTOP GENETICSHELPING YOU MANIPULATE GENOMES

LONDONEST. 2012BIOINFORMATICS TOOLS

GENE EDITING SERVICESBACKED BY LARGEST SEQUENCING PROVIDER

@EdwardPerello | @DesktopGeneticsWere a software company based in London and Boston

We help researchers manipulate genomes

You may be familiar with our public Deskgen software, used by 2000 users thousands of guides

We also provide a range of genomic services built with an advance internal version of our platform

We work with a number of organisations, some of whom are here today2

Edward Perello () - Keep it simple. Lay groundwork for complexity. Credential company.Victor Dillard () - you can now add Twist and Vectalys by the wayTWO PATHS TO DESKTOP GENETICSPRODUCTS & SERVICES DATA

@EdwardPerello | @DesktopGeneticsCRISPR genome editing is a multi step process, from guide design through to QC and data analysis

Many of the best practices require expensive, specialized equipment

Along with our partners, our vision is to enable you to do genome editing right from your computer3

Edward Perello () - This is what we sell.

All of this creates data.4TEACH AI TO PERFORM GENETIC SURGERYMACHINE INTELLIGENCE IS THE ONLY WAY TO EDIT THE GENOME SAFELY

@EdwardPerello | @DesktopGenetics

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Edward Perello () - The data feeds the machine.

INTRODUCE concept of AI for genomic surery.

INFORM people that AI is the only way to do genome surgeryAGENDA Brief intro to CRISPR

Our story so far

Taking CRISPR to the clinic

@EdwardPerello | @DesktopGenetics

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Editing the Genome with CRISPR

Desktop Genetics Ltd. 2016 An Illumina-backed company

@EdwardPerello | @DesktopGenetics

GENE THERAPY TACKLES DISEASESCRISPR is used to modify the genome of a patients cells

@EdwardPerello | @DesktopGenetics

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CRISPR OVERVIEWProgrammable two-component system

CAS9 NUCLEASERNACOMPONENT

@EdwardPerello | @DesktopGenetics

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CRISPR OVERVIEWProgrammable two-component system

CAS9 NUCLEASERNACOMPONENT

VARIABLE20 BP GUIDE RNACONSTANTREGION

@EdwardPerello | @DesktopGenetics

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CRISPR OVERVIEWProgrammable two-component system

CAS9 NUCLEASERNACOMPONENT

ACTIVE RNA-GUIDED CAS9 COMPLEX

@EdwardPerello | @DesktopGenetics

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CRISPR OVERVIEWMolecular find and replace for DNA

Source: McGovern Brain Institute Youtube Channel

@EdwardPerello | @DesktopGenetics

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Victor Dillard () - who is the audience? I feel like a nice comparison would be to put side by side the CRISPR system and a pair of scissors for example.

WHY EDIT GENOMES?Research & development Clinical interventions- Degenerative blindness- Custom disease models

- Humanization of heart valves- Swine fever resistance

- HIV eradicated in vitro- Immuno-oncology

- Clinical trials cured cancer - Clinical trials cured HIV

@EdwardPerello | @DesktopGenetics

WHY START A BUSINESS IN THIS SPACE?Huge biotechnology, life science and clinical prospects

75M PATIENTS3M RESEARCH EXPERIMENTS

@EdwardPerello | @DesktopGenetics

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Our Story

Desktop Genetics Ltd. 2016 An Illumina-backed company

@EdwardPerello | @DesktopGenetics

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2011 MEETING AT CAMBRIDGEMPHIL BIOSCIENCE ENTERPRISE DEGREE

@EdwardPerello | @DesktopGenetics

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2012 SYNTHBOT, CUTEC, SEEDTHE BIOTECH NESPRESSO

@EdwardPerello | @DesktopGenetics

2013 AUTOCLONE, TSB , RAISEHARDWARE OUT, SOFTWARE IN

@EdwardPerello | @DesktopGenetics2014 HORIZON CRISPR TOOL & TSBINTERNAL CRISPR R&D TOOL

@EdwardPerello | @DesktopGenetics

2014 - GUIDEBOOKCO-BRANDED CRISPR DESIGN & SALES PORTAL

@EdwardPerello | @DesktopGenetics

2015 DESKGEN, CLASS, PARTNERSUNITING & OPTIMISING OUR TOOLSET

@EdwardPerello | @DesktopGenetics

CRISPR EXPERIMENTS MODE, RICHER UI2015 DESKGEN, CLASS, PARTNERS

@EdwardPerello | @DesktopGenetics

2015 DESKGEN, CLASS, PARTNERSPARTNERSHIPS MAKE LITERAL DESKTOP GENETICS POSSIBLE

@EdwardPerello | @DesktopGeneticsTaking CRISPR into the clinic

Desktop Genetics Ltd. 2016 An Illumina-backed company

@EdwardPerello | @DesktopGenetics

INTERNATIONAL ENDEAVOURCRISPR is a global phenomenon

@EdwardPerello | @DesktopGeneticsControl set of essential genes2x depletion as significantBoth machine learning approaches beat two different human designed libraryOur combination approach is most effective on this set

2016 GUIDE PICKER, NGS, BEYONDMULTIDIMENSIONAL GUIDE SELECTION

@EdwardPerello | @DesktopGenetics

2016 GUIDE PICKER, NGS, BEYONDUSING BIOLOGICAL DATA TO TAKE OUR AI FURTHER

@EdwardPerello | @DesktopGeneticsHUMANS VS. MACHINESMACHINES OUT-PERFORM MANUAL DESIGN

CRISPR Success Rate

@EdwardPerello | @DesktopGeneticsControl set of essential genes2x depletion as significantBoth machine learning approaches beat two different human designed libraryOur combination approach is most effective on this set

28PERFORM GENETIC SURGERY WITH AIDesign CRISPR for every lab, teach our algorithms, go to the clinic

@EdwardPerello | @DesktopGenetics

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Edward Perello () - Speak aspirationally about the mission. Make them feel it.LIFE AT DESKTOP GENETICSRecognition in tech, biology, and everything in between

@EdwardPerello | @DesktopGeneticsStandardize and submit to GuideDB (API is incidental).

Edward Perello () - Remind them that its fun.

MENTION THE TEAM AND HOW GREAT THEY ARE.

Its not just a business.Victor Dillard () - add the LaBiotech award logo?WERE HIRINGJoin us in Shoreditch; tell your friends!

@EdwardPerello | @DesktopGenetics

Edward Perello () - We have a kickass office. We are going to have a lot more events.

We are ACTIVELY hiring

Mention the meetup I am hosting on the 14th.

TELL people to speak to people they think might be suitable for us.

INVITE people to speak with you.www.deskgen.comedwardp@desktopgenetics.com

@edwardperello

Desktop Genetics Ltd. 2015 Desktop Genetics Ltd. 2

@EdwardPerello | @DesktopGeneticsMEET UP ON 14 JULY see on FB page31