1. Rx.3 Cybele
Huntington„s: Disease in a Dish
Working Toward a Cure
December 10, 2013
Total interviews: 91
Contact: jharness@gmail.com
Team:
- Julie Harness
- Geeta Chauhan
- Hildegard Mack
- Nick Mordwinkin
- Jamieson Sheffield
Mentor: Antoun Nabhan

2. Team Cybele
Julie Harness, PhD
Postdoctoral Fellow, UCSF Department of Neurosurgery
Stem cell biologist with background in translational research
and Huntington’s Disease
Hildegard Mack, PhD
Postdoctoral Fellow, UCSF Department of Biochemistry
Biochemist and cell biologist with background in basic
research on aging and age-associated diseases
Geeta Chauhan
Director Innovation, Alcatel-Lucent CTO
New product development, Bell Labs Research
Translation, cloud computing, data analytics, multiple startups
Nick Mordwinkin, PharmD, PhD
Postdoctoral Fellow, Stanford
Stem cell biologist with background in next gen seq, drug
screening, cell therapy & cardiovascular disease modeling

3. The Problem: no cure for Huntington‘s Disease
Huntington‘s Disease (HD) at a glance



Deadly dominant genetic neurodegenerative disease affecting all age groups
1 in 10,000 Americans plus their families affected
Aberrant gene makes mutant protein, killing Medium Spiny Neurons (MSN)
One limitation on HD research/drug discovery


Lack of representative animal models and cell
models
Impossible to take patient samples since
affected cells are inaccessible and residing in
the brain
Our solution: Create affected cell type in a dish




Idea: reprogram somatic cells into iPSCs and
differentiate into MSNs
Starting material easily accessible
Reprogramming methods established
Efficient method to derive MSNs developed by us
differentiate
Human iPSC
MSN

4. Here is what we
thought….

5. Cybele’s MSN can add value in a multitude of ways
Tool for Basic Research and Drug Discovery
Discovery
Preclinical
Clinical Development
Translational “valley of death”
Ph 1
high
basic Target
Hit
throughput
research
screen
Ph 2
Ph 3
high Lead functional
First In Proof of NDA
Candidate
IND
content
assays
Human Concept
Investigational
New Drug
screen
New Drug
Application
Direct Therapeutic
Preclinical
Phase 1
Phase 2
Phase 3
FDA App &
Approval

6. Original Canvas Ideas

7. Here is what we did….

8. Questions to customers
Approach:
Q: Cells for in vitro use or direct therapeutic intervention or both?
A: Therapeutic is too expensive to finance directly given the small patient population
(not attractive to investors).
As an in vitro tool, it might be.
Product:
Q: Is it in vitro cultured cells/platform or data we generate from platform?
A: Platform. Academic researchers want to control their own experiment and pharma
wants to control compound libraries, experimental details and data.
* But they do outsource toxicity regularly.
Customer:
Q: Academic basic researchers or big pharma?
A: Both, but we will focus on them at different stages of development. We also add
tox screening companies and CRO‟s as important players.
Partnership ecosystem, licensing

9. Original Canvas Ideas

10. Cybele Biologics
Research Partners:
-Lisa Ellerby at Buck
-Bill Horn at USDA ARS
-Cyprotex
-other researchers/
startups we could
generate data and publish
with (e.g. Zephyrus)
Informal Partners:
-FDA – make them aware
of cells for preclinical
applica ons
Strategic Alliances:
-CHDI & other distributors
such as ATCC, Coriell, CDI
Supplier Rela onships:
-Lifetech (if we do their
reagents)
-Whoever we get iPS cells
from, maybe Coriell
Produc on of the
differen ated popula ons
Batch release valida on
Contac ng CROs, tox
companies, pharma etc
1 Enable in vitro research
in an indica on with
unmet medical need
(Hun ngton’s Disease)
Fundraising
2 Reduce drug discovery
costs by enabling
acquisi on of meaningful
data earlier in drug
discovery pipeline; fail
drugs earlier
IP for MSN differen a on,
freedom to operate
3 Reduce tox screening
costs – get meaningful
toxicity info early
CROs, startup
infrastructure, biotech &
pharma in bay area
-Overhead – space at QB3 or other incubator, regulatory body
approvals, costs of filing patent
-Cost of raw materials – undifferen ated cells from vendor and CRISPR
modifica on
-Differen a on of popula ons – hood rental, space at QB3, technician
-Batch release costs – verifica on, regulatory body approvals
Get:
Conference presence
Venture arms of pharma
Seminars in academia
Risk-free trial product
Google AdWords
Keep:
Cust serv/trouble shoot
Loyalty program
Grow:
Custom services
Assay Depot/Sci Exchange
CRO’s without exper se
to differen ate in-house
(e.g. System Biosciences)
Academics & NPOs
working on HD
Pharma/biotech
companies
(e.g. Lifetech)
Tox screening companies
(e.g. Cyprotex)
Distribu on by trusted
brand, like Lonza, Lifetech
or CDI
Nonprofit research orgs
like CHDI – recommend
product to collaborators
Direct sales of cellular popula ons
Direct sales of custom differen a on jobs to CROs
IPR Licensing (e.g. to CDI)

11. Here is what we
found….

12. Universe of Partners
Tools & Platforms
Pharma & Biotech
Cybele
NPOs

13. Two products to entice licensing/partnering with
cell supply companies and ADMET or pharma
 Cryopreserved cell
populations for direct sale
 Verstile for many downstream
applications
 New niche product in existing
market
* ADMET – Absorption Distribution
Metabolism Excretion Toxicity
 Functional assay: Multi Electrode
Array (MEA)
 New product in existing market with
infrastructure
 Ability to determine functional
response to compounds over long
period of time
 Ability to determine relative toxicity
of compounds in MSN

14. We identified several companies interested in each of our
product/service options as partners, customers and/or channels
Competitors
ATCC
Provide reagents
Joint
development
Joint
development
Velocity
System
Add to own products
Cells for sale as
research tool (off-theshelf and custom)
Cybele
Pfitzer
Functional
assays based on
our cells
Co-development /
Acquire
co-ownership
p
technology
Cyprotex
Color code: Channel partner Customer Partner
Competitors?
Competitors?
Drug candidate
developed using
our cells
CDI
GSK

15. Where to focus? Revenue Models Considered
• Drug discovery company: Potentially worth $ billions, but requires >$50M in
high-risk investment + significant capabilities added to existing MSN tech
• Cells: Relatively easy to advance to market, small revenue stream
• Screening services: Longer sales cycle than cells, but can be offered with
modest investment on top of that necessary to validate cell products
Product offering
# of customers
Revenue per
customer
Investment
Required and Exit
$2000 – 5000 per
customer per year
~$100k
(Viable)
Purified cell
products
•
•
•
>200
Mostly academic
Some pharma research
labs in HD
Screening projects
•
•
20-30
Pharma research labs w/
HD interest
$100k (pilot projects)
to $1M (large-scale
screens)
~$250k
(Viable)
Drug discovery w/
outlicensing of drug
candidates
•
•
10-15
Pharma companies with
licensing / acquisition
interest in HD
Potential for >$1B exit
event
$50-500M
(High-risk)

16. The market for Cybele as a tools company (I)
General considerations




Niche products in existing markets
Our cell product is a „consumable“
Most similar product : neuronal Progenitor. COG = $7, sales price = $1,200
Our cells: COG = $1,500, sales price = $2,200
Top-down analysis




Based on S1 of a similar company, CDI
Market for „cells for in vitro drug
discovery, toxicity testing and
chemical safety”
Assumes that HD share of purchases
reflects HD share of NIH budget
Target market would be entire SAM
What about a bottom-up estimate?
Target Market = SAM:
$8.5 million
TAM:
$3.5 billion

17. The market for Cybele as a tools company (II)
Bottom-up analysis

Customer segments considered
• Academic reserch labs in the US: ~ 250
• CROs specializing in neuronal cell toxicity: 1 validated


Products offered: Off-the-shelf cells, custom-derived cells, screening service with
partner CRO
Demand will be highly variable across customers
What we can get, based on
interviews
Type of service
What we would need for $1
Million/year
Profit
per each
Sales
per year
total
$500
220
$110,000
Custom cells
$10,000
10
$100,000
Screening service
$15,000
2
$30,000
Off-the-shelf cells
Total
Type of service
Off-the-shelf cells
Profit
per each
Sales
per year
$500
2,000
OR
Custom cells
$240,000
$10,000
100
OR
Screening service
$15,000
66

18. Timeline for Cybele as a tools company
2014
Q1
Q2
2015
Q3
Q4
Q1
Q2
net (revenue-costs), free samples
2016
Q3
Q4
Q1
Q2
net (revenue-costs), trial size sales
Q3
Cash Burn
(in $1,000)
80
60
40
20
0
-20
Partnerships Products
offered
Development
Milestones
-40
-60
Initi
alize
Beta product
Launch
Product refinement and assay development
product development and
upscaling production
New product lines
Further refinement and upscaling
Custom services
Cell sales
Free samples or sale of trial size
Research labs
CRO with expertice in ADMET analysis
Development of new products
Q4

19. The market for Cybele as a drug discovery company
General considerations





Use own cells for Drug discovery for HD, Risk mitigated due to use of hPSC
Result in refine cell products, new product lines
HD in NA + EY > 60,000
Onset age: 30-50 yr, 10% < 20yr, 10% > 55yr
OrphanDrug status, Tax breaks, FDA fee waiver, funding...
Top-down analysis





Drug Development cost ~ $100
Million
Price range $50k/patient/year $250k/patient/year
Target 50% market penetration
Low End Target Market = $1.5B
High End Target Market= $7.5B
TAM:
$3 Billion
SAM
$2.4 Billion
Target Market = $1.5B

20. Drug Discovery Business Model
•
•
•
•
•
•
Skill Sets/ Capabilities: Medicinal Chemistry, HTS, PKPD, Pharmacology
Use Partners and CROs
Exit by Phase 2a
Burn Rate ~ $1-1.5 Million / month
Phase 1 ~ 50 patients, Phase 2 ~ 100 patients, Phase 3 100-400 patients
Role Model Company: Evotec
Biological
Insight
Timeline
Cost
Key Partners
Lead
Optimization
12-18
months
18 months
$10-40 Mil
NCATS Assay, NCATS TRND
Screen,
Chemistry
groups
Preclinical
Phase1-2a
1 yr
2-3 yrs
$5 Mil
$15 Mil
NCATS
BrIDGS
Phase 3 to
Launch
$40 Mil
Velocity Pharmaceuticals,
CROs, Big Pharma

21. Cybele on the Investment Readiness Scale
IRL1
present new data to top five customers
review development plan data points
final definition of pre-clinical and clinical path
“proof of relevance” criteria/data/stages/quality
timeline to preclinical data and clinical data points
rough cost to each data point
value of program at each data point
non-dilutive money sources engaged
strengths/weakness compared to competition
probability and go/no go criteria at each stage
discussions with all pharma/large biotech
identify strategic need/fit at company level
present independent data
specific pre-clinical and clinical path/data needed
internal experts engaged
KOL discussions
Multiple pre-clinical and clinical paths identified
Independent data confirming base technology works
define clinical problem the technology solves
define early data customers consider gating
define best partners (like CROs) for outsourcing
quick check on IP
external experts identified
best outsourcing for this project
additional immediate data opportunities
IRL2
IRL3
IRL4
IRL5
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X
X

22. Here is what we will
do next….

23. Next Steps
1 Complete patent application.
2 Continue “bottom-up” market size analysis for cell product
1 Get back to the lab and create the data our interview partners would like
to see
2 Focus on licensing partners!

24. Immediate Next Steps: How much will they cost?
Cell
population
Total Cost ~
$10,000
Disease in
a dish
Running Total
Cost ~
$40,000
Functional
Assay
Running Total
Cost ~
$100,000
• Acquire new undifferentated iPS line
• CRISPR modify lines to contain varying repeats + control
• Repeat differentiation and QC in incubator lab
Interested Parties: Academic labs
• Provide batch to Buck Institute for independent
validation/collaboration on their phenotypic paper
• Refine protocol final stages for screening compatibility
• Quantify pathology in CRISPR-modified iPS differentiated cells
• Metabolism, cell adhesion, electrophysiology, survival
Interested Parties:
• Run HTS at Stanford HTBC – show cells are viable for HTS
• Generate MEA array platform for high content ephys screening
Interested Parties:
Contact: jharness@gmail.com

25. Big Thanks
• Teachers, Course Advisors, Mentors
• Karl Handelsman, Antoun Nabhan
• Customers and Industry KOLs who took time to do the interviews and
explain us things
• UCSF, QB3, Stanford, UC Berkeley for making the Entrepreneurship
resources available
• US Govt has many programs for small businesses, NIH is investing
greatly for Life Sciences Research Translation
• Lots of people ready to help. All you have to do is ask, and if one shows
passion and great drive people ready to line up and make your vision a
reality.

26. Appendix

27. Cybele
Business Model Canvas Progression

28. Cybele Biologics – Original Pre Course Canvas
Immediate:
-People to help protect
IP (lawyers etc.)
-Non-profits and
advocacy groups like
HDSA for the cause of
finding cure for a
specific genetic
disorder
Long term:
People to invest/help
fundraise/advise/strate
gize
Govt;
Research Universities
(IRBs)
Patient
Advocates, Support
Groups / Fund Raisers
-Production of
differentiated cellular
product
-Repeatable
process, efficient
delivery of cellular
lines, batch release QC
-Analytics tools for
creation/ testing /
validation
-Scientists creating
new cellular lines,
-Intellectual Property
on the diff cell lines
creation process
Immediate:
-Differentiated cell
populations – human
cellular model for HD
and other genetic
disorders
-Drug discovery
platform and tools
-Research conferences
(like CHDI)
-Lunchtime seminars @
universities
-Newsletters
-Direct outreach
-Social media (LinkedIn)
Reliable steady supply
Long term:
-Cellular
transplantation
therapies
-Future custom lines
for personalized
genomics
-Consent forms for
acquisition of new
materials
-Internet
-Direct Sales
-Partner w/trusted brand
and sell co-labeled
product through them
-StemBANCC
-Regional Support
Groups (eg HDSA)
Immediate:
-Drug Discovery
Researchers
-Pharma Companies
-Basic researchers
-iPSC Scientists
Long term:
-Patients
-Medical Centers
-Physicians
-CMS – what health
insurance co‟s will
reimburse
Ideal Customer now:
Doing drug discovery
for HD & other
neurodegenerative
disorders requires a
stead supply of the HD
Cellular Model for
testing
Attracting top scientists for creation of new cellular systems
Immediate:
-Sales of cellular models
Long term:
-Cell-based therapy
-Drug treatment mitigating/eliminating disease
Analytics + Simulation models to speed up creation of new lines
IPR Licensing
Production of the Cellular lines –
equipment, verification, regulatory body approvals

29. Cybele Biologics-Canvas version II (Week 1 Canvas)
Immediate:
-People to help protect IP
(lawyers etc.)
-Nn-profits and advocacy
groups like HDSA for the
cause of finding cure for
a specific genetic
disorder
Long term:
People to invest/help
fundraise/advise/strategi
ze
Govt;
Research Universities
(IRBs)
Patient
Advocates, Support
Groups / Fund Raisers
-Production of
differentiated cellular
product
-Repeatable
process, efficient delivery
of cellular lines, batch
release QC
-Analytics tools for
creation/ testing /
validation
-Scientists creating new
cellular lines,
-Intellectual Property on
the diff cell lines creation
process
-Consent forms for
acquisition of new
materials
An appropriate and
affordable cellular model
system for basic research
on HD.
An appropriate and in
large quantities
deliverable cellular
system for drug
discovery.
A direct therapeutic
agent in transplantation
therapies.
MVP needs to be
- Highly pure
- Batch-to-batch
homogeneity
- QC criteria tbd
- Producible in large
quantities
-Research conferences
(like CHDI)
-Lunchtime seminars @
universities
-Newsletters
-Direct outreach
-Social media (LinkedIn)
-Internet
-Direct Sales
-Partner w/trusted brand
and sell co-labeled
product through them
-StemBANCC
-Regional Support Groups
(eg HDSA)
Academic/Industry
researchers interested in
basic HD biology.
Drug discovery
companies with a focus
on HD or
neurodegenerative
diseases (may be
interested in expanding
to HD).
Companies developing
cell-based therapies (no
matter for which
disease, may be
interested in expanding
to HD)
HD Patients, their
physicians, medical
centers, CMS (what
health insurance co’s will
reimburse)
Attracting top scientists for creation of new cellular systems
Immediate:
-Sales of cellular models
Long term:
-Cell-based therapy
-Drug treatment mitigating/eliminating disease
Analytics + Simulation models to speed up creation of new lines
IPR Licensing
Production of the Cellular lines – equipment, verification, regulatory
body approvals

30. LaunchPad Centr al
Ke y Par tne r s
Ke y Activitie s
la wye rs (IP p ro t e ct io n ,
p ro d u ct ma n u fa ct u rin g
d ra ft in g lice n sin g
a g re e me n t s)
fu n d in g
a g e n cie s/fo u n d a t io n s wit h
a n in t e re st in su p p o rt in g
Week 3 Canvas
Value Pr op o sitio ns
Cu sto m e r
Re lation sh ip s
h ig h p u rit y ce ll p o p u la t io n
Ge t : p re se n t a t io n s a t
 CRO 's n e e d in g t o
fo r t h e ra p e u t ic
co n fe re n ce s (t a lk, p o st e r,
s u b co n t r a ct t o g e t
t ra n sp la n t s
ve n d o r e xh ib it io n )
r e lia b le , h ig h p u r it y
(R& D fo r n e w p ro t o co ls
h ig h p u rit y ce ll
Ge t : se min a rs a t
a n d ce ll lin e s)
p o p u la t io n s fo r
u n ive rsit ie s/re se a rch
co mp a n ie s wa n t in g t o
Hu n t in g t o n 's Dise a se
in st it ú t io n s
co mme rcia lize
p ro d u ct q u a lit y co n t ro l
p ro d u ct d e ve lo p me n t
HD re se a rch
cu st o me r su p p o rt
e st a b lish e d
ce ll p o p u la t io n s
ma rke t in g
re se a rch
b io p h a rma /b io t e ch
Ke y Re so u r ce s
Hu n t in g t o n 's Dise a se
Ge t : a d s in ma jo r scie n t ific
h ig h p u rit y ce ll
p o p u la t io n s fo r
t o t h e ir cu st o me rs,
Hu n t in g t o n 's Dise a se
d ist rib u t io n ch a n n e ls)
t h e ra p e u t ic e va lu a t io n
Hu n t in g t o n 's d ise a se
d ire ct sa le s (o rd e r via o u r
co mp a n ie s d e ve lo p in g
sh ip p in g t o cu st o me r)
re se a rch e rs in a ca d mia
p ro d u ct fo r fre e in re t u rn
re se a rch e rs e xp lo rin g
h o me p a g e , e xp re ss
g ra n t $
a n d in d u st ry (p ro vid e
t h e ra p ie s
jo u rn a ls ls
Ch an nea n d o n t h e se
co mp a n y (t o g e t a cce ss
la b sp a ce
Cu stom e r Se gm e n ts
Hu n t in g t o n 's Dise a se
a d viso ry b o a rd
t h e ra p e u t ics
co -la b e lin g wit h t ru st e d
IP
b ra n d (o rd e r a n d
Cu st o me r Ad vo ca cy
g ro u p s like CHDI, HDSA
d o in g b a sic re se a rch o n
we b p a g e /n e t wo rk)
To p Re se a rch Scie n t ist s
Co mp a n ie s/In st it u t io n s
d ist rib u t io n via t h e ir
fo r fe e d b a ck)
o t h e r Ne u ro d e g e n e ra t ive
d ise a se s like Pa rkin so n 's
Co st Str uctu r e
Re ve nu e Stre Co mme rce Su p p ly Ch a in
e am s
re n t fo r la b sp a ce
d ire ct sa le s
e q u ip me n t p u rch a se s
lice n sin g
fe e s fo r re g u la t o ry a p p ro va ls, IP-la wye rs
re a g e n t co st s
sa la rie s
ma rke t in g ca mp a ig n s
Li c e n se d fr o m b u si n e ssm o d e l g e n e r a t i o n .c o m u n d e r a Cr e a t i ve Co m m o n s At t r i b u t i o n -S h a r e Al i ke 3 .0 Un p o r t e d Li c e n se

31. Week 4 Canvas

32. Week 5 Canvas
Keep: Loyalty perks
Grow: Custom services

33. Week 6 Canvas

34. Cybele Biologics -- Week 7 Canvas - Discussion
KOL‟s
HDSA
CHDI
CIRM
QB3, SparkMed
NCATS
CDI
Lifetech
Cyprotex
Production of the
differentiated Cellular
lines
High purity pop. of
Repeatable
mature MSN
process, efficient delivery of
cellular lines,
High purity pop. of
committed MSN
Analytics tools for creation/ progenitors
testing / validation of
cellular lines
Well-char. Pop. of MSN
Athena Health
-ins co can influence
pharma to use our cells
FDA - influences
pharma to use our cells
for tox/whatever
Stanford hospital
Whoever Dr Layzer put
as contacts
Physiologically relevant
cell population of MSN
Get:
Conference presence
Venture arms of pharma
Seminars in academia
Risk-free trial product
Keep:
Cust serv/trouble shoot
Loyalty program
Grow:
Custom services
Well-char. populations
of other human cells
Scientists
creating new
cellular lines,
Intellectual
Property on the
diff cell lines
creation process
Assay Depot/Sci
Exchange
Cells w/native genotype
of known poly-Q length
Academics & NPOs
working on HD
Pharma Companies
Tox screening
companies
Distribution by trusted
brand, like Lonza
Reduction in drug
discovery costs
CRO‟s without
expertise to
differentiate in-house
Nonprofit research orgs
like CHDI – recommend
product to collaborators
Reduction in tox
screening costs
Production of the Cellular lines –
equipment, verification, regulatory body approvals
Attracting top scientists for creation of new cellular systems
Analytics + Simulation models to speed up creation of new lines
Direct sales of cellular populations
Direct sales of custom differentiation jobs to CROs
IPR Licensing

35. Cybele Biologics -- Week 7 Canvas
Research Partners:
-Lisa Ellerby at Buck
-Bill Horn at USDA ARS
-Cyprotex
-other researchers/
startups we could
generate data and publish
with (e.g. Zephyrus)
Informal Partners:
-FDA – make them aware
of cells for preclinical
applica ons
Strategic Alliances:
-CHDI & other distributors
such as ATCC, Coriell, CDI
Supplier Rela onships:
-Lifetech (if we do their
reagents)
-Whoever we get iPS cells
from, maybe Coriell
Produc on of the
differen ated Cellular lines
Repeatable process,
efficient delivery of cellular
lines,
Analy cs tools for
crea on/ tes ng /
valida on of cellular lines
Scien sts crea ng
new cellular lines,
1 Enable in vitro research
in an indica on with
unmet medical need
(Hun ngton’s Disease)
2 Reduce drug discovery
costs by enabling
acquisi on of meaningful
data earlier in drug
discovery pipeline; fail
drugs earlier
3 Reduce tox screening
costs – get meaningful
toxicity info early
Intellectual
Property on the
diff ell lines
crea on process
Produc on of the Cellular lines – equipment, verifica on, regulatory
body approvals
A rac ng top scien sts for crea on of new cellular systems
Analy cs + Simula on models to speed up crea on of new lines
Get:
Conference presence
Venture arms of pharma
Seminars in academia
Risk-free trial product
Google AdWords
Keep:
Cust serv/trouble shoot
Loyalty program
Grow:
Custom services
Assay Depot/Sci Exchange
CRO’s without exper se
to differen ate in-house
(e.g. System Biosciences)
Academics & NPOs
working on HD
Pharma/biotech
companies
(e.g. Lifetech)
Tox screening companies
(e.g. Cyprotex)
Distribu on by trusted
brand, like Lonza, Lifetech
or CDI
Nonprofit research orgs
like CHDI – recommend
product to collaborators
Direct sales of cellular popula ons
Direct sales of custom differen a on jobs to CROs
IPR Licensing (e.g. to CDI)

36. Cybele Biologics -- Week 8 Canvas
Research Partners:
-Lisa Ellerby at Buck
-Bill Horn at USDA ARS
-Cyprotex
-other researchers/
startups we could
generate data and publish
with (e.g. Zephyrus)
Informal Partners:
-FDA – make them aware
of cells for preclinical
applica ons
Strategic Alliances:
-CHDI & other distributors
such as ATCC, Coriell, CDI
Supplier Rela onships:
-Lifetech (if we do their
reagents)
-Whoever we get iPS cells
from, maybe Coriell
Produc on of the
differen ated popula ons
Batch release valida on
Contac ng CROs, tox
companies, pharma etc
1 Enable in vitro research
in an indica on with
unmet medical need
(Hun ngton’s Disease)
Fundraising
2 Reduce drug discovery
costs by enabling
acquisi on of meaningful
data earlier in drug
discovery pipeline; fail
drugs earlier
IP for MSN differen a on,
freedom to operate
3 Reduce tox screening
costs – get meaningful
toxicity info early
CROs, startup
infrastructure, biotech &
pharma in bay area
-Overhead – space at QB3 or other incubator, regulatory body
approvals, costs of filing patent
-Cost of raw materials – undifferen ated cells from vendor and CRISPR
modifica on
-Differen a on of popula ons – hood rental, space at QB3, technician
-Batch release costs – verifica on, regulatory body approvals
Get:
Conference presence
Venture arms of pharma
Seminars in academia
Risk-free trial product
Google AdWords
Keep:
Cust serv/trouble shoot
Loyalty program
Grow:
Custom services
Assay Depot/Sci Exchange
CRO’s without exper se
to differen ate in-house
(e.g. System Biosciences)
Academics & NPOs
working on HD
Pharma/biotech
companies
(e.g. Lifetech)
Tox screening companies
(e.g. Cyprotex)
Distribu on by trusted
brand, like Lonza, Lifetech
or CDI
Nonprofit research orgs
like CHDI – recommend
product to collaborators
Direct sales of cellular popula ons
Direct sales of custom differen a on jobs to CROs
IPR Licensing (e.g. to CDI)