Organ on a chip: A Commercialisation Report Card Trying to make an objective assessment on the status of a new technology (and avoid the hype)

Transcription

1 Organ on a chip: A Commercialisation Report Card Trying to make an objective assessment on the status of a new technology (and avoid the hype) Dr JM Wilkinson, Dr K Davidge Kirkstall Ltd. 06/12/2016

2 Content Challenges facing new technology introduction Hype vs reality Market need Key factors for tracking success Introducing a Report Card Format Trial run with status of some key players Recommendations for /12/2016

3 Some challenges for new technology New technology must meet a market need Winning grants is helpful but is not same as winning customers $ millions from VC s does not guarantee success (money does not change the laws of physics) Early engagement with customers is essential 06/12/2016

4 Hype Cycle for Emerging Technologies Organ on a chip has created high expectations Gartner, Inc. Stamford, CT USA

5 High expectations: organ on a chip will provide a more effective way to test safety and efficacy of new drugs There is a real market need! Could be a $ multi-billion market by 2020

6 Market opportunities in Drug Discovery Organ on chip goal is to replace HTS or animal 06/12/2016

7 Concept of Multi-Organ-Chip Edinburgh 2014 Materne 7

8 A Multitude of Approaches There is little standardisation Players are mainly start-up companies commercialising OOC prototypes developed in the universities Mainly plastic materials Different chamber and plate sizes Hurel, CN Bio, Mimetas use 96 well plate size TissUse and Kirkstall use 24 well plate size Wyss/Emulate has custom plates for each organ Pneumatic, Peristaltic, & Syringe pumps as well as gravity fed In general the control systems are more complex than the chips 06/12/2016

9 Organisation Chip Picture Controller Comments Each organ chip is developed by a different group so not clear how they will interconnect. Hurel (USA) MIT / CNBio (USA / UK) Mimetas (NL) Technology only available as a service from Hurel who now focus on delivery of ready seeded 24 and 96 plates Chip has in-built microfluidic pumps. Complex controller needed. CNBio offer as a service Chip is 96 well plate format. Works in a standard incubator with rocker plate Chip is size of cigarette Pack, chamber is 24 well plate size Control system uses Pneumatic pumping Chamber is 24 well plate format and in volume production with custom peristaltic pump

10 Key Requirements to replace animals Biological Physiologically relevant organoid models Connected organoids Long term culture /homeostasis Practical Easy to use and fast to set up Robust/repeatable No bubbles/blockages Scale 100 s animals/drug lead not 10,000 s as in HTS assays Economical Capital and consumable cost < animals Replacing HTS will require much lower cost 06/12/2016

11 Some Technical Challenges Obtaining representative human cells Media selection for co-culture of different cells Seeding cells inside microstructured chambers Getting adequate nutrient and oxygen to cells Connecting multiple chambers and flowing media Getting enough biological material for an accurate assay 06/12/2016

12 Cells for human on a chip? Animal cells may be easy to obtain and keep alive but they are not moving us forward Human tumour derived cell lines are easy to culture but are not representative of healthy tissue (basically cancer cells) ipsc (Induced Pluripotent Stem Cells) look promising but expensive and tough to grow Human Donor Tissue is gold standard but cryopreservation does compromise function 06/12/2016

13 Review Article : Organs-on-chips at the frontiers of drug discovery Esch, Bahinski & Huh, Nature Reviews Drug Discovery 14, (2015) Cancer cells Cancer cells Cancer cells Vascular cells Heart cells Cancer cells Liver cells Vascular cells Cancer cells Cancer Cells Cancer cells Liver Cells Why so many tumour derived cancer cells? They are tough to kill!

14 Given the right environment human cells can grow, differentiate and form organ-like structures

15 Report Card Format : Technical Factors Measure of progress Indicative Scores Physiologically relevant organoids Multiple organoids available Connected organoids Long term culture /homeostasis Easy to use / no bubbles / no leaks Robust/repeatable Scale to volume progress Economics

16 Report Card Format : Technical Factors Measure of progress Physiologically relevant organoids Indicative Scores 0 2D single cell type model 5 3D spheroid like model 9 functional differentiated organoid Multiple organoids available Connected organoids Long term culture /homeostasis Easy to use / no bubbles / no leaks Robust/repeatable Scale to volume progress Economics +3 liver +2 cardiomyocytes + 2 kidney +1 other organs 2 manual transfer of conditioned media 6 - flow of media between chambers 9 - recirculating flow of media 1 2 to 3 days 5 28 days 10 90days+ 2 in house only 5 available for external use / licence 9 already in use multiple sites 2 skilled postdoc researcher 5 fully trained lab technician 9 standard laboratory methods 1 single chambers 5 6 well chambers 7 24 well chambers 0 no cost data available 3 cost estimates available 7- on sale / cost match animals

17 Report Card Format : Commercial Factors Measure of progress Indicative Scores Organisation culture Market engagement Design for manufacture Ability to attract investment Sound business model Profitability TOTAL SCORE

18 Report Card Format : Commercial Factors Measure of progress Organisation culture Market engagement Design for manufacture Ability to attract investment Sound business model Profitability TOTAL SCORE Indicative Scores 2 - research laboratory 4 - research division in commercial organisation 8 - dedicated start up company 2 industrial partners in research consortium 4 sales to academics 6 sales to industrial innovators 9 sales to majority market 1 in house prototyping 4 industrial prototyping 6 industrial preproduction 9 - volume manufacture process +3 - research grants only +4 - Venture Capital +3 - Sales revenue 0 - no business plan 5 - CRO Research model only 7 - products only 9 - products and service 1 - burning investors capital 6 - break even 8 - profitable

19 Report Card : Example Technical Scores Measure of progress Indicative Scores W H C M T K Physiologically relevant organoids Multiple organoids available Connected organoids Long term culture /homeostasis Easy to use / no bubbles / no leaks Robust/repeatable Scale to volume progress Economics 0 2D single cell type model 5 3D spheroid like model 9 functional differentiated organoid +2 liver +2 cardiomyocytes + 2 kidney +1 other organs 2 manual transfer of conditioned media 6 - flow of media between chambers 9 - recirculating flow of media 1 2 to 3 days 5 28 days 10 90days+ 2 in house only 5 available for licence 9 already in use multiple sites 2 skilled postdoc researcher 5 fully trained lab technician 9 standard laboratory methods 1 single chambers 5 6 well chambers 7 24 well chambers 0 no cost data available 3 cost estimates available 7- on sale / cost match animals

20 Report Card : Example Commercial Scores Measure of progress Indicative Scores W H C M T K Organisation culture Market engagement Design for manufacture Ability to attract investment Sound business model Profitability 2 - research laboratory 4 - research division in commercial organisation 8 - dedicated start up company 2 industrial partners in research consortium 4 sales to academics 6 sales to industrial innovators 9 sales to majority market 1 in house prototyping 4 industrial prototyping 6 industrial preproduction 9 - volume manufacture process +3 - research grants only +4 - Venture Capital +3 - Sales revenue 0 - no business plan 5 - CRO Research model only 7 - products only 9 - products and service 1 - burning investors capital 6 - break even 8 - profitable

21 Overall Scorecard Result Name Technical score Technical ranking Commercial score Commercial ranking Wyss 31 #5 41 #1 Hurel 36 #3 39 #2 CNBio 30 #6 38 #3 Mimetas 32 #4 37 #4= TissUse 38 #2 29 #6 Kirkstall 40 #1 37 #4= Beware! Trial Scores only not really objective at this point

22 Commercial score 43 Leading Commercialistion? Leading Technical Approach? Wyss Hurel CNBio Mimetas TissUse Kirkstall Technical score

23 Commercial score Commercial & Technical Success Wyss Hurel CNBio Mimetas TissUse Kirkstall Technical score

24 Recommendations Refine the scoring criteria Add weighting factors? which criteria are more important Use some independent assessors Revisit scoring and ranking each year to track progress Thanks for your attention! 06/12/2016