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

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Edward Perello, CBO of Desktop Genetics, joins us at the Science: Disrupt London Session on Disruptor Stories to talk machine learning, CRSIPR, pivoting and his startup story.

Wissenschaft

@EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company
PERSONALISING CRISPR
GENOME EDITING
August 2016
Edward Perello| CBO & Founder

@EdwardPerello | @DesktopGenetics
DESKTOP GENETICS
HELPING YOU MANIPULATE GENOMES
LONDON
EST. 2012
BIOINFORMATICS
TOOLS
GENE EDITING
SERVICES
BACKED BY LARGEST
SEQUENCING PROVIDER

@EdwardPerello | @DesktopGenetics
TWO PATHS TO DESKTOP GENETICS
PRODUCTS & SERVICES  DATA

@EdwardPerello | @DesktopGenetics
4
TEACH AI TO PERFORM GENETIC SURGERY
MACHINE INTELLIGENCE IS THE ONLY WAY TO EDIT THE GENOME
SAFELY

@EdwardPerello | @DesktopGenetics
AGENDA
1. Brief intro to CRISPR
1. Our story so far
1. Taking CRISPR to the clinic

@EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company
Editing the Genome with
CRISPR

@EdwardPerello | @DesktopGenetics
GENE THERAPY TACKLES DISEASES
CRISPR is used to modify the genome of a patient’s cells

@EdwardPerello | @DesktopGenetics
CRISPR OVERVIEW
Programmable two-component system
CAS9
NUCLEASE
RNA
COMPONENT

@EdwardPerello | @DesktopGenetics
CRISPR OVERVIEW
Programmable two-component system
CAS9
NUCLEASE
RNA
COMPONENT
VARIABLE
20 BP GUIDE RNA
CONSTANT
REGION

@EdwardPerello | @DesktopGenetics
CRISPR OVERVIEW
Programmable two-component system
CAS9
NUCLEASE
RNA
COMPONENT
ACTIVE
RNA-GUIDED CAS9
COMPLEX

@EdwardPerello | @DesktopGenetics
CRISPR OVERVIEW
Molecular find and replace for DNA
Source: McGovern Brain Institute Youtube Channel

@EdwardPerello | @DesktopGenetics
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 PATIENTS
3M RESEARCH
EXPERIMENTS

@EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company
Our Story

@EdwardPerello | @DesktopGenetics
2011 – MEETING AT CAMBRIDGE
MPHIL BIOSCIENCE ENTERPRISE DEGREE

@EdwardPerello | @DesktopGenetics
2012 – SYNTHBOT, CUTEC, SEED
THE BIOTECH NESPRESSO

@EdwardPerello | @DesktopGenetics
2013 – AUTOCLONE, TSB , RAISE
HARDWARE OUT, SOFTWARE IN

@EdwardPerello | @DesktopGenetics
2014 – HORIZON CRISPR TOOL & TSB
INTERNAL CRISPR R&D TOOL

@EdwardPerello | @DesktopGenetics
2014 - GUIDEBOOK
CO-BRANDED CRISPR DESIGN & SALES PORTAL

@EdwardPerello | @DesktopGenetics
2015 – DESKGEN, CLASS, PARTNERS
UNITING & OPTIMISING OUR TOOLSET

@EdwardPerello | @DesktopGenetics
CRISPR EXPERIMENTS MODE, RICHER UI
2015 – DESKGEN, CLASS, PARTNERS

@EdwardPerello | @DesktopGenetics
2015 – DESKGEN, CLASS, PARTNERS
PARTNERSHIPS MAKE LITERAL DESKTOP GENETICS
POSSIBLE

@EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company
Taking CRISPR into the clinic

@EdwardPerello | @DesktopGenetics
INTERNATIONAL ENDEAVOUR
CRISPR is a global phenomenon

@EdwardPerello | @DesktopGenetics
2016 – GUIDE PICKER, NGS, BEYOND
MULTIDIMENSIONAL GUIDE SELECTION

@EdwardPerello | @DesktopGenetics
2016 – GUIDE PICKER, NGS, BEYOND
USING BIOLOGICAL DATA TO TAKE OUR AI FURTHER

@EdwardPerello | @DesktopGenetics
HUMANS VS. MACHINES
MACHINES OUT-PERFORM MANUAL DESIGN
CRISPRSuccessRate

@EdwardPerello | @DesktopGenetics
2
8
PERFORM GENETIC SURGERY WITH AI
Design CRISPR for every lab, teach our algorithms, go to the clinic

@EdwardPerello | @DesktopGenetics
LIFE AT DESKTOP GENETICS
Recognition in tech, biology, and everything in between

@EdwardPerello | @DesktopGenetics
WE’RE HIRING
Join us in Shoreditch; tell your friends!

@EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2015© Desktop Genetics Ltd. 2
www.deskgen.com
edwardp@desktopgenetics.com
@edwardperello

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

1. @EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company PERSONALISING CRISPR GENOME EDITING August 2016 Edward Perello| CBO & Founder

2. @EdwardPerello | @DesktopGenetics DESKTOP GENETICS HELPING YOU MANIPULATE GENOMES LONDON EST. 2012 BIOINFORMATICS TOOLS GENE EDITING SERVICES BACKED BY LARGEST SEQUENCING PROVIDER

3. @EdwardPerello | @DesktopGenetics TWO PATHS TO DESKTOP GENETICS PRODUCTS & SERVICES  DATA

4. @EdwardPerello | @DesktopGenetics 4 TEACH AI TO PERFORM GENETIC SURGERY MACHINE INTELLIGENCE IS THE ONLY WAY TO EDIT THE GENOME SAFELY

5. @EdwardPerello | @DesktopGenetics AGENDA 1. Brief intro to CRISPR 1. Our story so far 1. Taking CRISPR to the clinic

6. @EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company Editing the Genome with CRISPR

7. @EdwardPerello | @DesktopGenetics GENE THERAPY TACKLES DISEASES CRISPR is used to modify the genome of a patient’s cells

8. @EdwardPerello | @DesktopGenetics CRISPR OVERVIEW Programmable two-component system CAS9 NUCLEASE RNA COMPONENT

9. @EdwardPerello | @DesktopGenetics CRISPR OVERVIEW Programmable two-component system CAS9 NUCLEASE RNA COMPONENT VARIABLE 20 BP GUIDE RNA CONSTANT REGION

10. @EdwardPerello | @DesktopGenetics CRISPR OVERVIEW Programmable two-component system CAS9 NUCLEASE RNA COMPONENT ACTIVE RNA-GUIDED CAS9 COMPLEX

11. @EdwardPerello | @DesktopGenetics CRISPR OVERVIEW Molecular find and replace for DNA Source: McGovern Brain Institute Youtube Channel

12. @EdwardPerello | @DesktopGenetics 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

13. @EdwardPerello | @DesktopGenetics WHY START A BUSINESS IN THIS SPACE?Huge biotechnology, life science and clinical prospects 75M PATIENTS 3M RESEARCH EXPERIMENTS

15. @EdwardPerello | @DesktopGenetics 2011 – MEETING AT CAMBRIDGE MPHIL BIOSCIENCE ENTERPRISE DEGREE

16. @EdwardPerello | @DesktopGenetics 2012 – SYNTHBOT, CUTEC, SEED THE BIOTECH NESPRESSO

17. @EdwardPerello | @DesktopGenetics 2013 – AUTOCLONE, TSB , RAISE HARDWARE OUT, SOFTWARE IN

18. @EdwardPerello | @DesktopGenetics 2014 – HORIZON CRISPR TOOL & TSB INTERNAL CRISPR R&D TOOL

19. @EdwardPerello | @DesktopGenetics 2014 - GUIDEBOOK CO-BRANDED CRISPR DESIGN & SALES PORTAL

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22. @EdwardPerello | @DesktopGenetics 2015 – DESKGEN, CLASS, PARTNERS PARTNERSHIPS MAKE LITERAL DESKTOP GENETICS POSSIBLE

23. @EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2016 An Illumina-backed company Taking CRISPR into the clinic

24. @EdwardPerello | @DesktopGenetics INTERNATIONAL ENDEAVOUR CRISPR is a global phenomenon

25. @EdwardPerello | @DesktopGenetics 2016 – GUIDE PICKER, NGS, BEYOND MULTIDIMENSIONAL GUIDE SELECTION

26. @EdwardPerello | @DesktopGenetics 2016 – GUIDE PICKER, NGS, BEYOND USING BIOLOGICAL DATA TO TAKE OUR AI FURTHER

27. @EdwardPerello | @DesktopGenetics HUMANS VS. MACHINES MACHINES OUT-PERFORM MANUAL DESIGN CRISPRSuccessRate

28. @EdwardPerello | @DesktopGenetics 2 8 PERFORM GENETIC SURGERY WITH AI Design CRISPR for every lab, teach our algorithms, go to the clinic

29. @EdwardPerello | @DesktopGenetics LIFE AT DESKTOP GENETICS Recognition in tech, biology, and everything in between

30. @EdwardPerello | @DesktopGenetics WE’RE HIRING Join us in Shoreditch; tell your friends!

31. @EdwardPerello | @DesktopGenetics© Desktop Genetics Ltd. 2015© Desktop Genetics Ltd. 2 www.deskgen.com edwardp@desktopgenetics.com @edwardperello

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