Welcome to Day 2 of the Biotech fundamentals course, recap of day 1 learnings and overview of the day’s Agenda, covering: • Medical devices and diagnostics • Industrial applications and CleanTech • Aquaculture • Agriculture

1. Biotech Boot Camp
A biotechnology fundamentals course
Day 2
Presenters: Nick Weston + Viktoriia Hristova
Melbourne, Brisbane, Sydney
28 May - 18 June 2014

2. • Introduction
• Industrial applications and clean-tech
• Aquaculture and agriculture
• Medical devices and diagnostics
• Summary and wrap-up
Presentation Overview
Day Two
2

3. • Introduction
• Industrial applications and clean-tech
• Aquaculture and agriculture
• Medical devices and diagnostics
• Summary and wrap-up
Presentation Overview
Day Two
3

4. Biotechnology
4
Let’s recap from yesterday
A resilient sector that serves a diverse set of markets, across:
• Dx and medical devices
• agricultural products from animal health to seeds and crop
protection
• Rx (biomedical drugs)
• bio-based industrial products such as enzymes for industry
chemical processes and bio-remediation, bio-plastics and
bio-fuels (cleantech)

5. The term “life sciences" is used to connote sciences
concerned with the study of living organisms (as opposed
to physical sciences), including biology, botany, zoology,
microbiology, medicine, human therapeutics, veterinary
therapeutics, physiology, the ‘omics’ (eg: proteomics,
genomics), biochemistry, biotechnology, and related
subjects
Biotechnology
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Definitions

6. The term “medical devices and diagnostics" is used to describe any instrument,
apparatus, appliance, material or other article, and the software to run it,
intended by the manufacturer to be used for human beings to:
• diagnose, prevent, monitor, treat or alleviate disease, injuries or handicaps
• investigate, replace or modify the anatomy or a physiological process, or control
conception
and which does not achieve its principal intended action in or on the human
body by pharmacological, immunological or metabolic means, but which may
be assisted in its function by such means
Biotechnology
6
Definitions

7. The term “industrial biotechnology" is the application of
molecular biology technology to modify the biological
function of an organism to generate industrial products and
processes
• biomass-based materials such as fuels and chemicals; and
• the treatment of waste water and producing energy using more
efficient measures
Biotechnology
7
Definitions

8. The term “cleantech" recycling, renewable energy (wind
power, solar power, biomass and hydropower, biofuels),
information technology, green transportation, electric
motors, green chemistry, lighting, and many other
appliances that are now more energy efficient. It is a means
to create electricity and fuels with a smaller environmental
footprint and minimise pollution
Biotechnology
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Definitions

9. The term “agriculture" is the science, art, or practice of
cultivating the soil, producing crops, and raising livestock
and in varying degrees the preparation and marketing of
the resulting products
The term “aquaculture" is the cultivation of freshwater and
marine resources, both plant and animal, for human
consumption or use
Biotechnology
9
Definitions

10. The ‘Valley of Death’
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0

11. The ‘Valley of Death’
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1

12. Bioeconomy success
12 Source: Burrill Transforming Healthcare Report 2014, (originally in PwC Moneytree)
Strong global pipeline

13. Bioeconomy success
13 Source: Scientific American Worldview 2013
Australia No.7

14. Bioeconomy success
14 Source: PwC Bioforum Q1, 2014
Australia still strong for secondary raisings

15. Three criteria for a granted patent
• It must be novel
• It must be non-obvious
• It must have commercial utility
In Australia there are two types of patent applications
available
• Standard Patents
• Innovation Patents
Intellectual Property
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Patents

16. Utility Patent Protects the way in which an invention is made, how
it is used or how it functions
- Expires 20 years from the effective filing date
Design Patent Protects new ornamental design for an article of
manufacture
- Expires 14 years from the date of grant
Plant Patent Protects distinct and new plant varieties
- Expires 20 years from the effective filing date
Intellectual Property
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Patents

17. Intellectual Property
17 QUT-2004
Typical patent process and cost
$0
$50,000
$100,000
$150,000
$200,000
Provisional
Application
PCT
Application
National
Phase
Entry
Examination Renewals
Months
0 12 30
Patent granted
Cumulative cost
Defence
>$1m

18. Patents Trade Secrets
Protects against independent discovery
No protection against independent discovery
or reverse engineering
20 years of protection Protection lasts as long as secret
Scope for publication of development No opportunity for publication
Enforcement: infringement
Enforcement: breach of confidence, breach of
contract, breach of fiduciary duty
Protection can be broadened beyond
specific discovery/development
Protection specific to particular secret
Intellectual Property
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Patents v. Trade Secrets

19. The genomics era
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penicillins
sulphonamides
aspirin
psychotropics
NSAIDS
H2-antagonists
beta blockers
lipid lowerers
ACE-inhibitors
Biotech drugs
chronic
degenerative
disease associated
with ageing,
inflammation,
cancer
drugs against
targets identified
from disease genes
1900 20301950 1960 1970 1980 1990 2000 2010 2020 2040
NewTherapeuticCycles
1st generation 2nd generation 3rd generation
natural products
and derivatives
serendipity
receptors
enzyme
genetic engineering
cell pharmacology/
molecular biology
genomics/proteomics/metabolomics
Source: CMS, Lehman Brothers research

20. • Association of Molecular Pathology v Myriad Genetics
(2013)
• Naturally-occurring DNA is not patent eligible
• cDNA is patent eligible because it is not naturally-occurring
• Data protection
• 5 years Au, 6 years China, 10 years EU, 12 years US
• Hatch-Waxman Act extension (U.S.) (lesser of 5 years
from regulatory approval or 14 years of effective patent
life)
• Second medical use patents
Intellectual Property
Idiosyncrasies of protecting biologics - Patents
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21. Prescription medicine regulation
21
Recap
The TGA is accountable for the quality, safety, efficacy and
timely availability of drugs and medical devices in Australia
Performance targets for TGA drug evaluation and approval
were set and are included in the Regulations
The TGA operates on a 100% cost recovery basis

22. Prescription medicine regulation
22
Recap
Before a drug (prescription or non-prescription), medical
device, vitamin, nutritional, ‘TCM’, ‘alternative medicine’ or
herbal product can be marketed in, imported to, or exported
from Australia it must be listed on the Australian Register of
Therapeutic Goods (ARTG) by the TGA
New chemical entities and applications which require
expert advice are referred to the Advisory Committee on
Prescription Medicines (ACPM) for its non-binding
recommendations

23. Prescription medicine regulation
23
Recap
Also regulatory channels for
Biologicals
Biosimilars
Medical devices
IVD
GMOs (GT Register or licensing regime)

24. Basic overview of the life sciences
24
Recap
2003 2014
• Biotechs that wanted to emulate
bigpharma (a build model)
• Bigpharma wants to emulate
biotech (a buy model)
• Small molecule blockbusters • Antibody drug conjugates
• Cell based therapies
• Treatments that regulate or
alter genes
• Trial and error drug projects • Therapies designed to precisely
target molecular drivers
• Treatment of symptoms • Treatment of causes
• Move to curative medicine
• Human Genome Project
completed at cost of $3billion
• Illumina can sequence genome
for <$1,000

25. Typical life sciences development process
25 Graphic: Aptuit 2014
A life sciences biotech project starts with high throughput
screening identification of promising drug targets in the lab
(hits) that undergo in vitro testing for safety, specificity and
efficacy (candidate), proceeds to testing in animals and then
humans and ends in regulatory approval for marketing. The
focus of regulatory approval is on the safety and efficacy of the
drug in humans. Most countries have a government agency to
regulate and oversee the path to drug approval (approval).

26. Typical life sciences development process
26 Graphic: Aptuit 2014
Discovery
Disciplines
Development
Disciplines
Recap

27. Typical life sciences development process
27 Graphic: Aptuit 2014
Drug Design & Discovery
Preclinical Biosciences
API Development &
Manufacture
Solid State Chemistry
Pharmaceutical Sciences
Computational Support
Synthetic chemistry, small scale product synthesis
NMR, Chiral separation, UPLC/MSMS…..
Recap

28. Typical life sciences development process
28 Graphic: Aptuit 2014
Recap

29. Sector is approaching middle age
Hundreds of drugs and diagnostics approved
Technologies are proving their value (eg MaBs)
Biotech has plenty of scope for further innovation
Data analytics driving medical breakthroughs
Foundation built for substantial growth
Challenges remain
State of play
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Recap

30. • Introduction
• Industrial applications and clean-tech (morning tea)
• Aquaculture and agriculture
• Medical devices and diagnostics
• Summary and wrap-up (finished by lunch-time)
Presentation Overview
Today
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