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RNLAF 2016 03 McCauley DigBio

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Raymond McCauley

on 17 July 2016

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Transcript of RNLAF 2016 03 McCauley DigBio


New CRISPR Protein discovered
May break patent deadlock
UC Berkeley v. Broad Institute

DNA
Sequencing
+1-650-776-4561
for your attention
raymond@singularityu.org
Raymond McCauley
biochemistry
biophysics
electrical engineering
computer science
bioinformatics
nanotechnology
genetic engineering
DNA sequencing
personalized health
DISCLAIMER
These are my opinions and are not intended to reflect the views of any group or organization with which I may be affiliated.
Exponential Biosciences
systems
biology
past:
Biotech Chair: Singularity U
Co-Founder: Playtpus Project
Co-Founder & Chief Architect: BioCurious
Principal: Exponential Biosciences

Current Research Areas:
Personal Genomics
Therapeutic Genetic Engineering
Low Cost Access to Biotech Tools
Cancer Dx and Treatment
Singularity
University
Raymond McCauley
@raymondmccauley
Thank you
Raymond McCauley
Digital Biology:
Life is the New Black
raymond@singularityu.org
@raymondmccauley
+1-650-776-4561
Food
example: Okanagan Fruit Specialties
Not multinational, but family startup
Not implanted, but adjusted
Not labeled, but branded
2022
=
ApE = A Plasmid Editor
Rise of the
Biotech
Garage
Inventor
bioprinter project
OpenPCR
$599 DNA Copy Machine
+
Biohacking
Cow-Free
Milk & Cheese
Genetic
Engineering
Software Development
=
2015
=
Trends: Eco Foods, Food Security 3.0
Livestock Genotyping
trait prediction
breed, pedigree, parentage
identification
outbreak traceability
feed optimization
Genus PLC
and PIC, UK
For a copy of these slides,
biohacking resources for kids,
or other inquiries, email
raymond@singularityu.org
Subject:
Alltech & Digital Biology

Democratization
of
Biotechnology
How fast can you get to
"Hello World"
with 2 guys & a laptop in a garage?
A hackerspace for biotech

First biohacker lab in the world

Committed to making biotech
accessible, affordable, and open to everyone

Located in Sunnyvale, California

501c3 all-volunteer community lab, supported by monthly memberships and classes
Let's do that for biotech!
We teach
classes...
We mentor the
next generation
of scientists
and technologists
We support inventors
and entrepreneurs
BioCurious/FBI
Joint Conference on
Biohacking
June 12-16, 2012
More tools in more hands
=
More Breakthroughs
We are
everywhere
...
currently tracking
23 community labs
200+ individual labs
on 6 continents
You let
kids go hands-on
with blowtorches?!?
Why, yes!
Yes we do!
5 year old Harlan
makes Calcium burn
red
.
60+ companies out of BioCurious
129M+ in follow-on funding
Companies, universities copying model
Worldwide hackerspaces
Rise of the
Biotech
Garage
Inventor
Let's do that for biotech!
near-zero capital costs
outsourcing ecosystem
no IP taken
new players
new uses
new markets

$200 qPCR
20 min sample prep + readout
Matternet ONE
Boston
Paris
San Diego
Seattle
Oakland
Portland
Manchester UK
Biologigaragen
Biospace
Victoria, Canada
Denmark
Switzerland
Brooklyn
Sao Paolo
San Francisco
Shenzhen
Tokyo
Bio
Garage
Baltimore
Syn
Bio
Brasil
Also: Amsterdam, Berlin, Buenos Aires, Pittsburgh...
BGI,
Shenzhen
democratizing food
fruit by-products to nutrients, oils
Reading
DNA
Hacking
DNA
Writing
DNA
DNA Sequencing
Genetic Engineering
Systems Biology
Industrial Biology
Lab Automation
Outsourcing & Networks
Biotechnology
Renaissance
CRISPR/Cas9
CRISPR/Cpf1
TALEN
Swiss Army Knives for
Editing Genes
and regulatory regions
Knock-out
Knock-in
Repair
cheap
highly targeted
improvable
they work,
everywhere
New Genetic Engineering Tools
Clinical trial in
2017
to treat
Leber Congenital Amaurosis (CLA)
a rare form of blindness
announced Nov 4, 2015
reported Sept 25, 2015 in
Cell
Cfp1
image:
Cell
, Volume 163, Issue 3, p759–771, 22 October 2015

TALEN engineered T-Cells
"immune system transplant"
TALEN-based
Leukemia Treatment
announced Nov 5, 2015
Great Ormond Street Hospital, London
Sangamo Biosciences, 70 HIV treated, about half off ARVs
"What kind of infection?"
Test Types
Throat swab culture
DNA probe
RADT - Optical Immunoassay
RADT - Enzyme Immunoassay
Rapid qPCR
Time

48 hours
2 hours
<1 hour
<1 hour
<1 hour
Special Equip
-
$$
$$$
-
$
$200 qPCR
20 minute sample prep and run
First FDA Approval for
Direct-to-Consumer
Genetic Testing Company
late October 2015
Access to Tools
Biohacking
Resources
for Kids
Raymond McCauley
raymondmccauley
on Google Hangouts
or +1-650-776-4561
bit.ly/kidbiohack
"an app store for
DNA sequence"
+ Warburg Pincus + Sutter Hill Ventures = $100M
first partner:
Companies
Finding Cancer
with
Cell-Free DNA
partial list from Dr. Eric Topol
Home Lab
Tool Stack
microscope
incubator
PCR machine
gel electrophoresis
pipettor
workspace
refrigerator
freezer
water source
waste disposal
Garage Labs, Revisited
Now
Next
New
What's Different
cost
networks
outsourcing
international
DIYbio & biohackers v. traditional research
How we can help
Capabilities and Threats
bacteria/yeast/algae
new: agricultural
new: mammalian
recreational drugs
ag economic terrorism
BSL4 proliferation
Tech to Watch
Experimental Microbiomes
DNA Desktop Printers
Regulatory & Medical Tourism
CRISPR & Human Enhancement
RNA "Spray On" Genetic Engineering
Advanced Bionanotechnology
Cambrian
Genomics
Stem Cells /
Cell Replacement Therapy
Germline Genetic Engineering
Monsanto's RNA pesticides
DARPA's RNA vaccines
DNA Desktop Printers
Regulatory / Medical Tourism
GE & Human Enhancement
"Spray On" GE
DNA
Origami
RNA Interference
in spray form
pest control
delivery
regulation?
drought-resistant soy, approved Oct 2015
Argentina, Brazil, US = $3B
IPO imminent
real play: bioindustrials
photo credits: MIT Technology Review
Jar of RNA
was: $ million
now: $ hundreds
$5 = kill all beetles in an acre
Genetic Engineering
& Human Enhancement
Biofuels and Materials
Space Applications
microorganisms = factories
synthetic biology
industrial biology
industrial chemicals - food - agriculture biofuels - medical research
or
DNA = blueprints
new factories =
exponential
global capacity
61.4B gal
per year by 2018
source: Lux Research, Sept 2015
KLM Biofuel Program
Sustainability & Corporate Responsibility
80% CO2 reduction
Expensive
but getting cheaper
2013: 6 X kerosene
2016: 3 X kerosene
Fully sustainable in 2050 (SkyNRG)
& portable
Genetic Testing
& Military Strength
TBI
PTS
Depression
Find 30% most at risk
of PTS before combat
Charles Marmar, NYU
Cohen Veterans Center
genetic testing + imaging
screening
diagnosis
optimizing treatments
prognosis
to
cheaper
better
faster
networked
automated
tools
access
+
2001
=
+
+
Illumina Custom OmniExpress+ BeadChip
Assays about 1,200,000 oligo sequences
Precision Health
$149 - $249
The End of
Mammograms
Colonoscopies
&
?
circa 2018-2020
Next Generation Sequencing
Next
TALENs
Drag and Drop
Genetic Engineering
Transcription
Activator
Like
Effector
Nucleases
TALENs have modified genes in:
yeast
fruitfly
roundworm
cricket
zebrafish
frog
rat
pig
cow
thale cress
rice
silkworm
human somatic cells
pluripotent stem cells
Nature Reviews Molecular Cell Biology 14, 49-55 (January 2013)
http://www.brc.a-star.edu.sg/index.php?sectionID=12
hijacked from plant bacteria that genetically engineer their host plants
discovered by Bogdanove & Moscou, Iowa State University, in 2009
make double stranded breaks to knock-out and knock-in genes
Clustered
Regularly
Interspaced
Short
Palindromic
Repeats
hijacked prokaryotic immune system mechanism
discovered by Horvath & Barrangou, DuPont/Danisco, in 2010
immunize industrial bacteria
gene silencing in stem cells
CRISPRs
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali, Luhan Yang, Kevin M. Esvelt, John Aach, Marc Guell, James E. DiCarlo, Julie E. Norville, and George M. Church
Science 15 February 2013

Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong, F. Ann Ran, David Cox, Shuailiang Lin, Robert Barretto, Naomi Habib, Patrick D. Hsu, Xuebing Wu, Wenyan Jiang, Luciano A. Marraffini, and Feng Zhang
Science 15 February 2013
Academically (for reference)...
Commerically...
$43 million Series A , Nov 2013
Flagship Ventures
Polaris Partners
Third Rock Ventures
Partners Innovation Fund
TALENs
CRISPRs
Human
Infertility
Prenatal Genetic Dx
Mitochondrial Therapy
Germ-Line Therapy
Enhancement
- Disease Risk
+ Intelligence
+ Longevity
NOW
NEXT
genetic
engineering
BGI, Shenzhen, China
Sequence geniuses
Find ~2000 markers
Use to score humans
Use to pick embryos by IVF
+1-2 sigma (15-30 IQ points)
Use to engineer embryos by CRISPR
CELSS
Closed Environment
Life Support Systems
Rockets, then Biotechnology
Radiation
Hardening
Resource
Utilization
Microgravity
Adaptation
Solugen
peroxide
from CO2
with enzymes
20x cheaper
portable
Dual-Use Technologies
Lego
+ DNA Origami
= Nanotechnology
DNA
Origami
MSTN
LRP5
APP
CCR5
PCSK9
FUT2
PCSK9
SLC30A8
Lean muscles
Extra-strong bones
Alzheimer’s-resistant/immune
HIV-resistant/immune
Lower coronary risk
Stomach flu resistant
Heart disease
Type 2 Diabetes
Genes to Knock Out
adapted from George Church, private communication
Behavioral and Mental Traits
MAOA gene & aggression
last to implement
poorly understood
Therapeutics,
then
Enhancement
Gene Expression
"gene doping"
EPO, red blood cell production
Myostatin, muscle growth inhibition
VEGF, blood vessel production
IGF-1, targeted muscle mass increase
redneck
genetic
engineering
threats
Full transcript