Restoring Sight
Treatment Options
Currently available:
Vitamin A
Has not shown consistent benefits
in clinical evaluation
In clinical trials:
Retinal Prosthesis
Vision limited to few pixels
1. Algae
Optogenetic
Treatment for RP
2. Gene therapy
injection
Light-sensitive genes
are extracted
3. Light mediation
Light is shone
directly into eyes
Re-grow rods +
cones in retina
5. A Better Life
Genes are added to
patient’s DNA
Competitive Analysis
Vitamin A
Retinal
Prosthesis
Optogenetics
Invasiveness
Non-invasive
Invasive surgery Simple
outpatient
injection
Efficacy
No consistently Patients see
proven efficacy only fuzzy
shapes
Genes
repaired,
photoreceptors
regrown
Patient
Population Size
2M
worldwide
100,000 in
US
10,000 in
US with
profound
visual
impairmen
t
Pricing
Benchmarks
Retinal
Prosthesis for $60K-$100K
RP
Ranibizumab
for macular $48K
degeneration
Target market size: 10,000 x $60,000 = $600 Million
Bottom Up Market Size Analysis
150 retinal degeneration specialists
treat RP patients in the US
600 existing patients per specialist
10% of these patients have severe
genetic form of RP
$60,000 cost per therapeutic
package
US target market size: $540 million
Revenue Model
Patient
User
Therapeutic
Package
$$$
Purchaser
Payer
Partners & Allies
Intellectual Property
Grants
$$$
IP
Clinical Trials
$$$
Data, FDA
Approval
$$$
Equity
Core Competency:
Research + Development
$$$
Manufacturers
Gene
Therapy
Serum
Optical
Device
$$$
Venture
Capital
Key Stakeholders
RETINAL SPECIALISTS
PAYERS
RP PATIENTS
Value proposition:
increased clinical efficiency
Value proposition:
increased cost
effectiveness
Value proposition:
Effective treatment
Key activities:
•publish papers
•sales team
•Retinal centers &
conferences
Key activities:
•Clinical trial design
addresses cost
effectiveness outcomes
•Hire experience
reimbursement VP
Key activities:
•RP communities &
foundations
•eye clinics & centers
•internet & social media
Timeline
Pre-Animal Research
Safety & Tolerability Research
Animal Research
today
year 1
Preclinical Research
Phase I Phase II Phase III
Clinical
year 2
year 3
year 4
year 5
Market
launch
Major Risks
Technology
Regulatory
Regulatory
Market
Development
Pre-Clinical
Clinical
Market
Market
Funding
Milestone
Major Expenses
Amount
Source
Proven animal
efficacy
Licensing / Hiring/
R&D
$3.5MM
Philanthropic grants
SBIR
Angel funding
Venture funding
Preclinical safety
demonstrated
Pre-Clinical studies
$4MM
Venture Funding
Philanthropic grants
SBIR
Human trials
completed
Phase I,II,III trials,
regulatory
submission
$15MM
Venture Funding
NIH grants
Positive cashflow
Establish sales and
distribution force
$3MM
Venture Funding
Major Expenses
Development
Patent and Licensing Costs
5 patents * ~40K = ~$200K
Hiring the Initial Team
3 yrs * (CEO+CSO) = ~1MM
3 yrs * (key hires) = ~1.5MM
Pre-clinical
Trial Contractors
$400K (pre-clinical) + $50K
(animal)
Research Lab Upkeep
3 yrs * $250K = $750K
Clinical Trials
$4000/patient * 200 = $800K
Clinical
Sales / Marketing
50 reps * $50K = 2.5MM
Initial Core Team
Founders
CEO
Chief Scientific Officer
Qualification: Proven biotech
leader, led multiple companies
from inception to exit
Qualification: Pioneer in field of
optogenetics
Example:
Ralph Kauten, MBA
Example:
Dr. Karl Deisseroth,
MD, PhD
Scientific Advisory Board
Experts in:
Example
Gene therapy
Eg. Dr. Richard Mulligan, Harvard Gene Therapy Initiative
Optogenetics
Eg. Dr. Ed Boyden, Chief, MIT Synthetic Neurobiology Group
Retinal ophthalmology
Eg. Dr. John Pach, Mayo Clinic
Key Early Hires
VP of Regulatory
Affairs
VP of R&D
VP of
Reimbursement
Legal consultant
Qualification:
Track record of
success with FDA
PMA and RAC
applications
Qualification:
Expert in
development of
protein biology
systems
Qualification:
Track record of
success in
reimbursement of
ophthalmologic
products
Qualification:
Leading biotech
law firm
Example:
Barbara Niksch,
MBA
Example:
Jennie Lill, PhD
Example:
Stuart Raetzman
Example:
Wilson Sonsini
Goodrich Rosati
Conclusion
Mission:
Restoring vision to RP
patients who currently
lack a solution
Market:
US market $700-800M
World market $2.1-2.4B
Risk:
Significant technical and
regulatory risk; partially
mitigated by risk benefit
profile for RP
Future:
Platform technology with
wide IP coverage, has
spectrum of promising
therapeutic applications
Acknowledgements
Teaching Team
Professor Tom Byers
Thomas Haymore
Kurt Sandholtz
Mentor
Dr. Jason Davies, UCSF Neuroscience
Ophthalmologists
Dr. Mark Blumenkranz, Chairman of
Stanford Ophthalmology
Dr. Joyce Liao, Stanford Ophthalmology
Dr. Theodore Leng, Stanford
Ophthalmology
Optogenetic Researchers
Dr. Raag Airan, Johns Hopkins Radiology
Dr. Polina Anikeeva, Stanford
Bioengineering
Remy Durand, Stanford Bioengineering
Funding and Business Strategy Experts
Dana Mead, Partner, KPCB
Dr. Beth Seidenberg, Partner, KPCB
Oleg Nodelman, Portfolio Manager,
Biotechnology Value Fund
Intellectual Property Experts
Gregg Kyle, Senior Associate, Office of
Technology Licensing
Trevor Roubison, Associate, Office of
Technology Licensing
Neurosurgeons
Dr. Jamie Henderson, Stanford
Neuroscience
Dr. Paul Larson, UCSF Neuroscience
Drug Discovery Experts
Dr. Dr. Solow-Cordero, Manager of
Stanford High Throughput Bioscience
Center
Dr. Kevin Grimes, Director of SPARK
Other Physicians
Dr. Homero Rivas, Stanford Bariatric
Surgeon
Dr. Graham Creasey, Stanford Professor
of Spinal Cord Medicine
Appendix
Global Market Size
$1800 MM
Global market
$600 MM US
market
New patient market size
150 retinal degeneration specialists
treat RP patients in the US
15 new patients per specialist per
year
10% of these patients have severe
genetic form of RP
$60,000 cost per therapeutic
package
US target market size: $14 million