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Team 11: Recombinant Gammaglobulin 
The first pure antibody source for patients who can’t make their 
own antibodies ($7 billion existing market). 
We interviewed 163 people to test our business hypothesis. 
Matt Spindler 
Principal Investigator 
PhD UCSF 
Postdoc Stanford 
Specialty is immune 
therapies 
Copyright © 2014 GigaGen, Inc. 
David Johnson 
C-Level 
MBA UC Berkeley 
PhD Stanford 
Specialty is genomics 
innovation 
Jennifer Keller 
Industry Expert 
MBA UC Irvine 
MS UC Berkeley 
Specialty is clinical 
marketing
Copyright © 2014 GigaGen, Inc. 
Situation 
• Millions of sick people can’t make their own antibodies, or they make faulty 
ones 
• To stay healthy, these people need to get antibodies from another source 
• Now, plasma is collected from thousands of healthy people and fractionated to 
isolate antibodies 
• The antibodies are then shot back into the sick people 
• This is called gammglobulin, or IVIG: a $7 billion market! 
1000’s of people 
Plasma collection Plasma fractionation IVIg
Copyright © 2014 GigaGen, Inc. 
Complication 
Demand exceeds supply: 
•Three major shortages in the past 15 years 
•Strong market growth and off-label use will 
continue to stress the supply 
•Serum collection will hit upper limit 
Quality problems: 
•Factor XIa contaminants disrupted supply in 2010 
and caused significant morbidity 
•IgA contamination can cause anaphylactic shock 
•Batch-to-batch variation in genetic content limits 
efficacy 
•No ability to engineer isotype or genotype content 
for particular applications 
IVIg Shortages 
Year
Solution: Recombinant Gammaglobulin 
Remember these guys? 
If nature doesn’t have enough, make 
it yourself! 
•More predictable 
•More engineerable 
•More scalable 
Copyright © 2014 GigaGen, Inc.
Get out of the building? Get out of the country! 
• Would anyone use recombinant? 
• Who is the first target patient population 
• Why would they use recombinant? 
• Who would we need to work with to make 
the product? 
• What are the regulatory risks? 
• What would clinical trials look like? 
Copyright © 2014 GigaGen, Inc.
Finding the Key First Customers 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Cancer patient 
Autoimmunity 
patient 
Infectious disease 
doctor 
Oncologist
Customers Teach Us What They Value 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Cancer patient 
Autoimmunity 
patient 
Infectious disease 
doctor 
Purity 
Differentiating 
preclinical and 
clinical work 
“Hyperimmune” 
targeting specific 
pathogens 
Oncologist
Learning: Customer Value Proposition 
Peace of 
mind 
Convenience 
Consistency 
Copyright © 2014 GigaGen, Inc. 
Unreliable 
supply 
Viral 
contamination 
Live freely 
Survive 
Blood protein 
contamination 
rhIVIg production 
Home 
infusion 
Consisten 
t product 
Price 
uncertainty 
In vitro 
In vitro 
production 
Clear 
reimbursement Renewabl 
e resource 
Clinical 
studies 
Infusions 
every 3rd 
week 
Longer ½ 
life in vivo 
Don’t 
get sick 
Save 
money 
Feel healthy
Which Stakeholders Are Most Important? 
Copyright © 2014 GigaGen, Inc. 
Healthcare professionals 
Patients 
Pharmas 
Payer/Regulatory 
Distribution
Learning: Stakeholder Importance 
Infectious disease 
pediatric 
Biotechs 
w/innovation 
programs 
Copyright © 2014 GigaGen, Inc. 
Healthcare professionals 
Patients 
Pharmas 
Immunologists 
IDF 
Baxter/Grifols/CSL 
Payer/Regulatory 
Distribution 
autoimmunity PID 
Medicare/private payer CBER 
Wholesale Specialty pharma 
Less value More value
Learning: Stakeholder Importance 
Infectious disease 
pediatric 
Biotechs 
w/innovation 
programs 
Copyright © 2014 GigaGen, Inc. 
Healthcare professionals 
Patients 
Pharmas 
Immunologists 
IDF 
Baxter/Grifols/CSL 
Payer/Regulatory 
Distribution 
autoimmunity PID 
Medicare/private payer CBER 
Wholesale Specialty pharma 
Less value More value
Business Model Canvas: Partners 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Infectious disease 
doctor 
Purity 
Differentiating 
preclinical and 
clinical work 
“Hyperimmune” 
targeting specific 
pathogens 
IDF 
CBER
Learning: Ecosystem for Development 
• Yeast library protocol development 
internal vs. external 
• Small-scale vs. large-scale cGMP 
production 
• cGMP vs. pilot production 
• US vs. Europe vs. Asia production 
Copyright © 2014 GigaGen, Inc.
Business Model Canvas: Protein Partners 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Infectious disease 
doctor 
Purity 
Differentiating 
preclinical and 
clinical work 
“Hyperimmune” 
targeting specific 
pathogens 
IDF 
CBER 
Small scale 
GMP (EU) 
Large scale 
GMP (EU) 
Protein library 
protocol 
development 
Protein library 
protocols
Business Model Canvas: Channels 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Infectious disease 
doctor 
Purity 
Differentiating 
preclinical and 
clinical work 
“Hyperimmune” 
targeting specific 
pathogens 
IDF 
CBER 
Small scale 
GMP (EU) 
Large scale 
GMP (EU) 
Protein library 
protocol 
development 
Protein library 
protocols 
Distributer 
Specialty pharma 
Hospitals 
GPO
Learning: Distribution Models 
Copyright © 2014 GigaGen, Inc. 
Sp Pharmacy 
patient 
Distributer 
Lg Hosp Pharm 
GPO 
(Manages pricing for Sp 
P/Pharm) 
Greatest 
volume Some 
volume 
Very little 
volume
Business Model Canvas: Channels 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Infectious disease 
doctor 
Purity 
Differentiating 
preclinical and 
clinical work 
“Hyperimmune” 
targeting specific 
pathogens 
IDF 
CBER 
Small scale 
GMP (EU) 
Large scale 
GMP (EU) 
Protein library 
protocol 
development 
Protein library 
protocols 
Distributer 
Specialty pharma 
Hospitals 
Group purchasing 
organization
Learning: Ecosystem for Development 
Resources Activities Partners 
<100pt study for launch 
Copyright © 2014 GigaGen, Inc. 
Physician channels 
for clinical studies 
Regulatory Consultant 
Recombinant Ig library 
IP protecting in vitro 
production process 
Microfluidic chip designs 
Pre-clinical assays SOPs 
IDF 
Clin Study sites (PID) 
Med Ad Board 
GMP production 
facilities 
Human Blood Source 
Build rIg library 
pipeline 
Pre-clinical studies for 
IND 
Informatics for library 
QC Microfluidic chip foundry 
GLP rodent CRO 
Distribution 
Specialty Pharmacy or 
Wholesaler
Copyright © 2014 GigaGen, Inc. 
Conclusion: Next Steps 
Path to Revenues, OP Plan, Cust 
Data points 
High Fidelity MVP 
Business Case Defined 
Left-side of the Canvas 
Right-side of the Canvas 
Product/Market Fit 
Problem/Solution validation 
Low Fidelity MVP, Clinical Paths 
ID’d 
First Pass Canvas 
Verdict on Phase II Grant: 
•NCI Phase I is a separate program from 
IVIG 
•Definitely moving forward on that 
separate program 
•Separate program supports big pharma 
partnering 
Investment Readiness Verdict: 7 
•MVP is our big hurdle 
•Financing with pending Phase I SBIR and 
profit from two big pharma deals 
•Looking for $15-20MM Series A in 
2015
Post-Script: Hyperimmune Directed IVIG 
Copyright © 2014 GigaGen, Inc. 
Primary immune 
deficiency patient 
Immunologist 
Infectious disease 
doctor 
Purity 
Differentiating 
preclinical and 
clinical work 
“Hyperimmune” 
targeting specific 
pathogens 
IDF 
CBER 
Small scale 
GMP (EU) 
Large scale 
GMP (EU) 
Protein library 
protocol 
development 
Protein library 
protocols 
Distributer 
Specialty pharma
Business Model Canvas: Week 1 
Copyright © 2014 GigaGen, Inc.
Business Model Canvas: Week 3 
Copyright © 2014 GigaGen, Inc.
Business Model Canvas: Week 5 
Copyright © 2014 GigaGen, Inc.
Business Model Canvas: Week 6 
Copyright © 2014 GigaGen, Inc.

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Gammaglobulin I-Corps@NIH 121014

  • 1. Team 11: Recombinant Gammaglobulin The first pure antibody source for patients who can’t make their own antibodies ($7 billion existing market). We interviewed 163 people to test our business hypothesis. Matt Spindler Principal Investigator PhD UCSF Postdoc Stanford Specialty is immune therapies Copyright © 2014 GigaGen, Inc. David Johnson C-Level MBA UC Berkeley PhD Stanford Specialty is genomics innovation Jennifer Keller Industry Expert MBA UC Irvine MS UC Berkeley Specialty is clinical marketing
  • 2. Copyright © 2014 GigaGen, Inc. Situation • Millions of sick people can’t make their own antibodies, or they make faulty ones • To stay healthy, these people need to get antibodies from another source • Now, plasma is collected from thousands of healthy people and fractionated to isolate antibodies • The antibodies are then shot back into the sick people • This is called gammglobulin, or IVIG: a $7 billion market! 1000’s of people Plasma collection Plasma fractionation IVIg
  • 3. Copyright © 2014 GigaGen, Inc. Complication Demand exceeds supply: •Three major shortages in the past 15 years •Strong market growth and off-label use will continue to stress the supply •Serum collection will hit upper limit Quality problems: •Factor XIa contaminants disrupted supply in 2010 and caused significant morbidity •IgA contamination can cause anaphylactic shock •Batch-to-batch variation in genetic content limits efficacy •No ability to engineer isotype or genotype content for particular applications IVIg Shortages Year
  • 4. Solution: Recombinant Gammaglobulin Remember these guys? If nature doesn’t have enough, make it yourself! •More predictable •More engineerable •More scalable Copyright © 2014 GigaGen, Inc.
  • 5. Get out of the building? Get out of the country! • Would anyone use recombinant? • Who is the first target patient population • Why would they use recombinant? • Who would we need to work with to make the product? • What are the regulatory risks? • What would clinical trials look like? Copyright © 2014 GigaGen, Inc.
  • 6. Finding the Key First Customers Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Cancer patient Autoimmunity patient Infectious disease doctor Oncologist
  • 7. Customers Teach Us What They Value Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Cancer patient Autoimmunity patient Infectious disease doctor Purity Differentiating preclinical and clinical work “Hyperimmune” targeting specific pathogens Oncologist
  • 8. Learning: Customer Value Proposition Peace of mind Convenience Consistency Copyright © 2014 GigaGen, Inc. Unreliable supply Viral contamination Live freely Survive Blood protein contamination rhIVIg production Home infusion Consisten t product Price uncertainty In vitro In vitro production Clear reimbursement Renewabl e resource Clinical studies Infusions every 3rd week Longer ½ life in vivo Don’t get sick Save money Feel healthy
  • 9. Which Stakeholders Are Most Important? Copyright © 2014 GigaGen, Inc. Healthcare professionals Patients Pharmas Payer/Regulatory Distribution
  • 10. Learning: Stakeholder Importance Infectious disease pediatric Biotechs w/innovation programs Copyright © 2014 GigaGen, Inc. Healthcare professionals Patients Pharmas Immunologists IDF Baxter/Grifols/CSL Payer/Regulatory Distribution autoimmunity PID Medicare/private payer CBER Wholesale Specialty pharma Less value More value
  • 11. Learning: Stakeholder Importance Infectious disease pediatric Biotechs w/innovation programs Copyright © 2014 GigaGen, Inc. Healthcare professionals Patients Pharmas Immunologists IDF Baxter/Grifols/CSL Payer/Regulatory Distribution autoimmunity PID Medicare/private payer CBER Wholesale Specialty pharma Less value More value
  • 12. Business Model Canvas: Partners Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Infectious disease doctor Purity Differentiating preclinical and clinical work “Hyperimmune” targeting specific pathogens IDF CBER
  • 13. Learning: Ecosystem for Development • Yeast library protocol development internal vs. external • Small-scale vs. large-scale cGMP production • cGMP vs. pilot production • US vs. Europe vs. Asia production Copyright © 2014 GigaGen, Inc.
  • 14. Business Model Canvas: Protein Partners Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Infectious disease doctor Purity Differentiating preclinical and clinical work “Hyperimmune” targeting specific pathogens IDF CBER Small scale GMP (EU) Large scale GMP (EU) Protein library protocol development Protein library protocols
  • 15. Business Model Canvas: Channels Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Infectious disease doctor Purity Differentiating preclinical and clinical work “Hyperimmune” targeting specific pathogens IDF CBER Small scale GMP (EU) Large scale GMP (EU) Protein library protocol development Protein library protocols Distributer Specialty pharma Hospitals GPO
  • 16. Learning: Distribution Models Copyright © 2014 GigaGen, Inc. Sp Pharmacy patient Distributer Lg Hosp Pharm GPO (Manages pricing for Sp P/Pharm) Greatest volume Some volume Very little volume
  • 17. Business Model Canvas: Channels Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Infectious disease doctor Purity Differentiating preclinical and clinical work “Hyperimmune” targeting specific pathogens IDF CBER Small scale GMP (EU) Large scale GMP (EU) Protein library protocol development Protein library protocols Distributer Specialty pharma Hospitals Group purchasing organization
  • 18. Learning: Ecosystem for Development Resources Activities Partners <100pt study for launch Copyright © 2014 GigaGen, Inc. Physician channels for clinical studies Regulatory Consultant Recombinant Ig library IP protecting in vitro production process Microfluidic chip designs Pre-clinical assays SOPs IDF Clin Study sites (PID) Med Ad Board GMP production facilities Human Blood Source Build rIg library pipeline Pre-clinical studies for IND Informatics for library QC Microfluidic chip foundry GLP rodent CRO Distribution Specialty Pharmacy or Wholesaler
  • 19. Copyright © 2014 GigaGen, Inc. Conclusion: Next Steps Path to Revenues, OP Plan, Cust Data points High Fidelity MVP Business Case Defined Left-side of the Canvas Right-side of the Canvas Product/Market Fit Problem/Solution validation Low Fidelity MVP, Clinical Paths ID’d First Pass Canvas Verdict on Phase II Grant: •NCI Phase I is a separate program from IVIG •Definitely moving forward on that separate program •Separate program supports big pharma partnering Investment Readiness Verdict: 7 •MVP is our big hurdle •Financing with pending Phase I SBIR and profit from two big pharma deals •Looking for $15-20MM Series A in 2015
  • 20. Post-Script: Hyperimmune Directed IVIG Copyright © 2014 GigaGen, Inc. Primary immune deficiency patient Immunologist Infectious disease doctor Purity Differentiating preclinical and clinical work “Hyperimmune” targeting specific pathogens IDF CBER Small scale GMP (EU) Large scale GMP (EU) Protein library protocol development Protein library protocols Distributer Specialty pharma
  • 21. Business Model Canvas: Week 1 Copyright © 2014 GigaGen, Inc.
  • 22. Business Model Canvas: Week 3 Copyright © 2014 GigaGen, Inc.
  • 23. Business Model Canvas: Week 5 Copyright © 2014 GigaGen, Inc.
  • 24. Business Model Canvas: Week 6 Copyright © 2014 GigaGen, Inc.

Editor's Notes

  1. Think about adding a personal story to the setup here…jenn was talking to some doctors about the technology and got the suggestion of ivig rather than oncology Also add a set up on the technology
  2. Who uses gammaglobulin?
  3. Put the person first here
  4. Didn’t really understand why anyone uses recombinant. We wanted to hear what patients had to say. Turns out what they think is actually pretty different from what doctors and pharmacists have to say. Patients do care a lot about possible contamination. This stuff keeps them healthy, and they take it forever.
  5. Talk about how IDF links together everything.
  6. Talk about how IDF links together everything.
  7. Talk about how IDF links together everything.
  8. Talk about how production partners help you get over the hurdle you need to get over to get ANYWHERE. Before we started, we didn’t understand who does what, and where, in the protein production industry. We certainly weren’t going to go out and build a 10,000 liter reactor ourselves.
  9. We learned through our travels that we needed to go to EU for small and large scale GMP, because of their unique experience there with large scale protein production in yeast. We would need to build our own protocols for protein libraries. Nobody has ever done this before, so it must be a technical focus. It’s also a huge opportunity that builds value in the company.
  10. But how would we get our product to the customers? Drugs get to customers in pretty complicated ways. We had a few hypotheses about how this might work for us.
  11. This was one of the hardest areas to get information. It’s a real complex system, and very few people appear to comprehend the full system. So we really needed to connect the dots! Talk about Mike – because he understands the the system.
  12. We found that distributers and specialty pharma are really the way to go.But we still need to learn a lot more about how we should get our product to market, and how to get it paid for.
  13. By the time we finished 163 interviews, we discovered that there was a whole ecosystem that we could leverage through the entire process, from protein production to clinical studies to human blood sample collection. The process has helped us understand who to talk to, how much things will cost, and what will be the timelines to achieve particular tasks.
  14. We are going hard a partnering at JP Morgan in January We are going hard at raising Series A in 2015 We are going hard at following our Phase I grants with Phase II and executing strong on our Phase II grants.
  15. Infectious Disease doctors were not all that excited about a recombinant IVIG replacement But, they kept telling us that there is an opportunity to make specific pathogen-directed targeted IVIG We will be speaking with dozens of stakeholders over the next month to explore whether this should be product 1!!