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Rapid, cost-effective, and scalable manufacturing technologythat uses tobacco plants as biofactories of therapeutics
“insero” = to plant ”gen” = gene
I-Corps Contacts: 52
“Using tobacco to produce a life-enhancing treatment for emphysema disease”
Team Inserogen
Mentor Vasilis Voudouris, Ph.D., MBA
Managing Director, Vereniki Solutions
Lucas Arzola Ph.D. Candidate, Chemical
Engineering Emphasis in Biotechnology
Entrepreneurial Lead
Principal InvestigatorKaren McDonald, Ph.D.
UC Davis Professor, Chemical Engineering
1st Prize, 2010 Big Bang! Business Plan Competition at UC Davis
Elevator Pitch Award, 2011 UC Berkeley B-Plan Competition
E-Team Grant, National Collegiate Inventors and Innovators Alliance
25+ years of experience in Plant Made Proteins Research
Inventor in 1 issued patent and 4 patent applications
20 years of experience in Process Development Manufacturing and Strategic Planning
Animal and human vaccines
Personalized medicine/biosimilars
Proteins for research use and diagnostics
Industrial enzymes for bioenergy
Cosmetic and cosmeceutical
Nutraceutical/ functional food
A Green Multi-Product Manufacturing Platform
Harvested tobacco
> 90% reduction in capital costsReduction in operating costsLarge scale production in weeks, not months
The Business Model Canvas Target Product – seasonal & pandemic flu vaccines
Wholesalers
Tobacco Suppliers
Gene Synthesis Comp.
CMO
CRO
FDA
Intellectual Property
Manufacturing Facility
R&D
Manufacturing
Regulatory Approval
Marketing
Speed
Cost-Effectiveness
Scalability
Safety
Customization
U.S. Based Supply
Publications
Conferences
Long Term Contracts
Government
HHS BARDA
DOD DARPA
Vax Manufacturers
NGOs
Contract Manufacturing
Fully Integrated Manufacturing (Sales)
Licensing (Royalties)
CDC
Facility Investment
Manufacturing Costs
IP Licensing Costs
Marketing Costs
Version 1
The Business Model Canvas
Speed
Cost-Effectiveness
Scalability
Safety
Customization
U.S. Based Supply
Version 1
What we thought: these features can meet a need for the influenza vaccine market.
Target Product – seasonal & pandemic flu vaccines
Getting Out of the Laboratory!What we did: Contacted vaccine industry stakeholders
What we found:
Pandemic flu2B
Surge capacity Opportunity
DOSES
Seasonal flu
YEAR 1
Seasonal fluSeasonal flu250M
YEAR 2 YEAR 3
Unpredictable market Flu vaccines are commodities Large companies are not willing
to change their manufacturing platform
“This is a billion-dollar investment we are not willing to make”
PIVOT TO THERAPEUTIC M
ARKET
The Business Model Canvas Target Product – seasonal & pandemic flu vaccines
alpha-1 antitrypsin (AAT)
Tobacco Suppliers
CRO
FDA
Alpha-1 Foundation
CMO
Patients
Physicians
Pulmonary Labs
Large Biotech Comp.
Conferences
Long Term Contracts
Publications
Wholesalers
Direct to Patient
Speed
Cost-Effectiveness
Scalability
Safety
Reliable Supply
Intellectual Property
Manufacturing Facility
R&D
Manufacturing
Regulatory Approval
Marketing
Gene Synthesis Comp.
Government
Facility Investment
Manufacturing Costs
IP Licensing Costs
Marketing Costs
Grants
Upfront & Milestone Payments (from Partners)
Lean Biotech Company (Sales)
Licensing (Royalties)
Alpha-1 Project Customization
U.S. Based Supply
Version 2
What are we selling?
First target product: Alpha-1 antitrypsin (AAT)
Indication: AAT Deficiency (AATD) – hereditary disorder resulting in low levels of AAT in lungs, leads to emphysema, often before age 40
Less than 10% of AAT Deficiency individuals have been properly diagnosed
Worldwide Estimates 3.4 million with AATD 116 million carriers
“AAT may be one of the most common serious hereditary disorders in the world” - President, Alpha-1 Europe
AAT Treatment is Expensive There is no known cure for AAT Deficiency Treatment: Augmentation therapy – weekly AAT infusions
5 g AAT per patient per week Annual: 260 g AAT per patient
Cost per patient: ~$100,000/year for life
Competitor Pricing Prolastin - $350/gram Zemaira - $430/gram Aralast - $510/gram Alfalastin - $450/gram
Value Proposition
Value proposition: We can provide recombinant AAT (we’ve already produced it in the lab!)
Reliable and scalable supply Safer production Cost reduction – we believe we can substantially reduce treatment cost
Problem: Commercial AAT for augmentation therapy is derived from donated human blood Cost Reliable Supply: “There are problems with supply.
We need new ways of making AAT.” – Scientific Director, Alpha-1 Foundation
Safety: “The risk of transmission of infectious agents cannot be completely eliminated.” – Prescribing information for Zemaira
Target Market
6,000 patients
AAT deficiency in the United States
100,000 patients
Millions of patients
Under treatment
Total
New indications for AATDiabetes, cystic fibrosis, others
$600M
$10B
Billions
Cost per patient: $100,000/year
“As diagnosis improves and new indications are found, we will need more AAT produced.” – Scientific Director, Alpha-1 Foundation
Business Model and Customer Archetype
Lean biotech business model
+
Core technology Outsource to CMO Finished Drug Product
“Starting out and for as long as you can, be a lean biotech company.” – CEO, Biotech Company
Patent protection
(for clinical development) Customer: Established biotech company Interested in blood proteins and/or alternative
manufacturing platforms Looking to increase their pipeline, gain market share Hundreds of millions (not billions) in revenue Hundreds (not thousands) of employees
Customer Decision Making
• 400-500 opportunities per year• 1-2 weeksInitial Evaluation
• 80-100 opportunities• 2-3 monthsDetailed Analysis
• Internal innovation committee meeting• Once a monthGo – No Go Decision
• 25-30 opportunities• 2-3 monthsDue Diligence
• Agree to terms, business development team approval• 2-3 monthsBusiness Discussions
• Establish work plan and define milestones• Project startsDeal is signed
Total time: 6-9 months
How do customers decide if they want our product?
Revenue Model
= money= information
= relationship
Large Pharma
INFLUENCER
Hospital/Clinic
Physicians
Patient
Employer
Government
Taxpayer
Government Payor
Private Payor
Pulmonary Function Lab
Wholesalers
= AAT
The Business Model Canvas Target Product – seasonal & pandemic flu vaccines
alpha-1 antitrypsin (AAT)
Tobacco Suppliers
CRO
FDA
Alpha-1 Foundation
CMO
Patients
Physicians
Pulmonary Labs
Large Biotech Comp.
Conferences
Long Term Contracts
Publications
Wholesalers
Direct to Patient
Speed
Cost-Effectiveness
Scalability
Safety
Reliable Supply
Intellectual Property
Manufacturing Facility
R&D
Manufacturing
Regulatory Approval
Marketing
Gene Synthesis Comp.
Government
Facility Investment
Manufacturing Costs
IP Licensing Costs
Marketing Costs
Grants
Upfront & Milestone Payments (from Partners)
Lean Biotech Company (Sales)
Licensing (Royalties)
Alpha-1 Project Customization
U.S. Based Supply
Version 3
Next Steps
FundingAlpha-1 Project
Venture Philanthropy Grant
NSF SBIR Phase I Program
IP Licensing agreement of core technology
Freedom to Operate analysis
Business Team Development & Incorporation
Join Engineering Translational Technology Center