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What is CRISPR?
A guide to what CRISPR is and its far-reaching
applications in healthcare, agriculture, & more.
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J O I N T H E C O N V E R S A T I O N O N T W I T T E R
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Ja Lee
Ja Lee is an intelligence analyst at CB Insights, where she
produces data-driven analysis and research reports on trends
in biotechnology and medical devices.
Prior to joining CB Insights, Ja worked with clinical trials at
Children's National Medical Center. She is a graduate of the
University of North Carolina at Chapel Hill with majors in
Biology and Spanish. She received her Master's degree in
Physiology from North Carolina State University.
@Ja_Lee2 | jlee@cbinsights.com
Intelligence Analyst
A B O U T T H E A N A L Y S T
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Contents
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What is CRISPR?
How does it work?
Startups to watch
Applications & limitations
What lies ahead
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CRISPR ushers in a new wave of gene editing
G E N E E D I T I N G 2 . 0
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CRISPR leads among other gene editing techniques
CRISPR gene
editing
technology leads
against older
methods.
Before there was CRISPR,
there were other gene-editing
techniques known as Zinc
Finger Nucleases
(ZFNs) and Transcription
Activator-Like Effector
Nucleases (TALENs).
CRISPR
TALENs
ZFNs
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CRISPR-Cas9 system has 3 main players
C R I S P R = C L U S T E R E D R E G U L A R L Y I N T E R S P A C E D S H O R T P A L I N D R O M E R E P E A T S
1 Guide RNA
aka "GPS coordinates"
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3
Cas9 protein
aka "molecular scissors"
DNA
containing the sequence
we want to insert
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How does CRISPR work?
B R E A K I N G C R I S P R D O W N
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Understanding How The CRISPR Gene-editing
Process Works
W H A T I S C R I S P R ?
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Understanding How The CRISPR Gene-editing
Process Works
W H A T I S C R I S P R ?
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Understanding How The CRISPR Gene-editing
Process Works
W H A T I S C R I S P R ?
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Understanding How The CRISPR Gene-editing
Process Works
W H A T I S C R I S P R ?
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Understanding How The CRISPR Gene-editing
Process Works
W H A T I S C R I S P R ?
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More CRISPR startups are on the horizon
K E Y S T A R T U P S
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Incumbents begin CRISPR clinical trials in 2018
Funding: $163M
Select investors: Google Ventures,
Khosla Ventures, Third Rock
Ventures
Clinical trial focus: Genetic eye
disease Leber Congenital
Amaurosis type 10 phase 1
Funding: $162M
Select investors: Versant
Ventures, Celgene, Bayer
Clinical trial focus: Beta-
thalassemia (Europe) & sickle
cell disease (US) phase 1/2 trials
Funding: $85M
Select investors: Atlas Venture,
Novartis Venture Funds, OrbiMed
Advisors
Clinical trial focus: Transthyretin
Amyloidosis (Pre-clinical)
T O P 3 P U B L I C C O M P A N I E S
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" it might be better to describe CRISPR not as
scissors but as a Swiss army knife, a tool with a
panoply of functionalities that all stem from the
action of a single molecular machine."
Jennifer Doudna, PhD
A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution
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Mammoth Diagnostics provides a CRISPR-based
platform for disease detection.
It uses a simple paper-based test in which a liquid
sample (e.g. saliva) is applied and subsequently, a
color change becomes visible. A smartphone app
can be used to upload that data to the cloud, in
which an analysis is delivered in 30 minutes.
Images: Mammoth Diagnostics
N E W A P P L I C A T I O N : A T - H O M E D I A G N O S T I C S K I T
Disclosed Funding:
$120K
Key team members:
Select Investors:
NFX Guild
Trevor Martin
Co-Founder, CEO
Jennifer Doudna
Co-Founder, Chair of Scientific
Advisory Board
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Beam Therapeutics develops therapeutics
using a more precise method of CRISPR
called base-editing. By tethering a modified
CRISPR enzyme and an editing enzyme
together, this technology allows editing to
occur without cutting into the DNA at all.
Images: Beam Therapeutics
P R E C I S I O N G E N E T I C M E D I C I N E : B A S E - E D I T I N G
Disclosed Funding:
$87M
Key team members:
Select Investors:
ARCH Venture Partners,
F-Prime Capital
David Liu, PhD
Co-Founder
Feng Zhang, PhD
Co-Founder, Board Member
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CRISPR will spur cross-industry innovation
A P P L I C A T I O N S & L I M I T A T I O N S
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Faster
Cheaper
Precision medicine
Drug discovery process could be transformed
Image:
P H A R M A & B I O T E C H
Current process
With CRISPR
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CRISPR could transform food supply & quality
F O O D & A G R I C U L T U R E
April 2016
1st CRISPR-edited organism to bypass USDA
The white button mushroom, edited to not
brown as quickly, bypassed USDA regulations.
Sept 2017
Another USDA approval
Yield10 Bioscience's CRISPR plant
Camelina sativa with enhanced omega-3 oil
bypasses regulations.
October 2017
Leaner livestock
Scientists at the Chinese Academy of
Sciences in Beijing used CRISPR to
genetically engineer pig meat with
24% less body fat
Jan 2018
CRISPR saving chocolates
Innovative Genomics Institute at Berkeley
is working with candy company Mars to
genetically engineer cacao plants
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Immune system complications
Yet, there are still some hurdles ahead
L I M I T A T I O N S
Mosaic generation
Off-target activity
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2
3
Different cutting enzymes
(molecular scissors)
Edit stem cells in egg or sperm
cells
Use a different Cas protein or
ex-vivo therapies to bypass
immune system
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What lies ahead for CRISPR
C O N T R O V E R S I E S & F U T U R E O F C R I S P R
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Designing-your-own-
baby
Germline
modifications
CRISPR applications present ethical concerns
C O N T R O V E R S I E S L I E A H E A D
Bringing back extinct
species
Biohacking
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Xenotransplantation
Future applications of gene-editing
Images: eGenesis; E. OTWELL AND M. TELFER, ScienceNews.org; DARPA
F U T U R E O F C R I S P R
Gene drive
Genome editing as a weapon of mass
destruction?
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Questions?
Twitter: @ja_lee2
jlee@cbinsights.com
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