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Bio- and Nanotechnology:
Emerging Medical Miracles onthe Horizon
Marsha K. Millonig, MBA, RPhPresident & CEO
Catalyst Enterprises, LCC
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Todays Objectives
Understand biotechnology andnanotechnology
Describe the biotechnology market size
Explain what fields are parts of the emergingbusiness of life science
List a number of new therapies resulting from
bio and nanotechnology Discuss the implications of these new
therapies on pharmacy and its technology
partners
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Why is it Important?
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Global Population Growth
Earths capacity is estimated at 12 billion people
It took 100 million years before Earth had 1 billionpeople in 1830
Yet only 170 years for the population to reach 6billion plus
2005: 6,436,562,930
2009: 6,752,560,109 the people who have ever lived are on theplanet today
Less than 100 years before we reach capacity
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Biotechnology is needed to
Create better fuels that dont harmenvironment
Create tools to clean environment, feeda burgeoning global population, cureuntold human suffering
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Biotechnology Definition
Use of cellular and biomolecular processes to solveproblems or make useful products. Lifesciencesbiology/chemistry technology affectingdiscovery and development of products for:
Healthcare (therapeutics, diagnostics, drug delivery, cell andgene therapy, devices, drug/device combinations)
Agriculture (food, feed, fibers, transgenics)Industrial and Environment (reduce pollution, clean energy)
All driven by a new set of enabling technology(genomics, combinatorial chemistry, SNPs, proteomics,sequencing)
The firsts
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Development is evolutionary
4000-2000 BC: biotechused to leaven bread &ferment beer
1830: Proteins discovered1833: First enzyme
discovered
1865: Genetic science
begins-Mendel discoverslaws of heredity
1879: Chromosomesdiscovered
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Development is evolutionary
1906: The term genetics introduced
1919: The word biotechnology is first
used in print
1938: The term molecular biology coined
1941: The term genetic engineering isfirst used
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Development is evolutionary
1953: Watson and Crick DNAStructure
1958: DNA made in test tube
Sickle cell caused by AA change1960: Messenger RNA discovered1967: First automatic protein
sequencer is perfected1969: Enzyme synthesized in
vitro the first time1970: First enzyme discovered to
cut DNA molecules at aspecific site
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Development is evolutionary
1971: First complete synthesis of a gene1973: First time DNA fragments linked1975: First monoclonal antibodies made1976: First NIH research guidelinesBoyer co-founds Genentech, 1st bio co.1978: Recombinant insulin first produced
1980: Oil-eating microbes patented by Exxon1982: First recombinant DNA vaccine for livestock1983: First whole plant grown from biotechnology
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Development is evolutionary
1980: First gene-synthesizersdeveloped
1981: First transgenic animals1982: First biotech drug: insulin
1983: First artificial
chromosome synthesizedFirst genetic markers for
inherited disease found
http://www.google.com/imgres?imgurl=http://www.drug3k.com/img/humulin_15702_2.jpg&imgrefurl=http://www.drug3k.com/drug/humulin-N-15702.htm&h=114&w=109&sz=3&tbnid=VD1Hk6MjiVw40M::&tbnh=87&tbnw=83&prev=/images%3Fq%3Dhumulin%2Bn%2Bpicture&hl=en&usg=__BTvvqEV7pMDKh3UkeJBMcrrEjgA=&sa=X&oi=image_result&resnum=1&ct=image&cd=17/28/2019 Millon i g Asap 12009 Partial
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Development is evolutionary
1984: DNA fingerprinting developed
1985: Genetic fingerprinting entered
as evidence in courtroom1986: Interferon first anti-cancer drug
from biotech
First genetically engineered vaccinefor humans: Hepatitis B
Microbes used to clean oil spill
http://www.google.com/imgres?imgurl=http://www.idfpr.com/DPR/images/fingerprint.gif&imgrefurl=http://www.idfpr.com/DPR/employer.asp&h=351&w=291&sz=6&tbnid=SxUWalALBXUJAM::&tbnh=120&tbnw=99&prev=/images%3Fq%3Dfingerprint%2Bpicture&hl=en&usg=__TfpKbPbu4rUadOSDEMeKv2kBKgs=&sa=X&oi=image_result&resnum=3&ct=image&cd=17/28/2019 Millon i g Asap 12009 Partial
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but speeding up.
1988: First US patent for genetically alteredanimala transgenic mouse
1989: First DNA exoneration now 216 (4/08)
1990: First food product from biotechapproved: modified yeast
1994: First FDA approval for first whole foodproduct: FLAVRSAVR tomato
1997: First weed & insect resistant cropsdeveloped
First cloned animal: Hello Dolly!
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but speeding up.
1998: Human embryonic stem cells linesestablished
Herceptin approved-considered firstpharmacogenomic (personalized) medicine
First complete animal genome: roundworm
2000: First complete map of a plant genomeFirst draft human genome
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but speeding up.
2004: First genetically modified pet: theGloFish
FDA clears genotyping test to aid inmedication selection
First cloned pet, a kitten2005: Skin cells converted to embryonic stem
cells
1 billionth acre biotech seed planted
First complete dog genome: boxer
2006: FDA approves Gardasil-first vaccine forcancer-causing virus
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but speeding up.2007: Successful reprogramming human skin cells
to create cells indistinguishable from embryonicstem cells
Biotech cattle that cannot develop prions=no mad
cow disease2008: draft corn genome
10 disease bearing stem cell lines created
Mature human embryos created from adult skin
cells: personalized stem cells for diseasetreatment
First synthetic genome: the road to creating life
First complete map of cancer patient genome
First US FDA application for gene-therapy/cancer
http://www.google.com/imgres?imgurl=http://barfblog.foodsafety.ksu.edu/HappyCow.jpg&imgrefurl=http://barfblog.foodsafety.ksu.edu/articles/raw-milk/&h=550&w=440&sz=228&tbnid=c5blb_wVTYomZM::&tbnh=133&tbnw=106&prev=/images%3Fq%3Dcow%2Bpicture&hl=en&usg=__e20AqAXLkb5aX2t5D00tXouURRE=&sa=X&oi=image_result&resnum=2&ct=image&cd=17/28/2019 Millon i g Asap 12009 Partial
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2005 and Today 2005: Biotechnology will transform industries, including
health care
Today: Biotechnology IS transforming industries
Industrial convergence of farmers, doctors, drugmakers,chemical processors, computer and communicationcompanies, energy companies and many others into the
business of life science. Is revolutionizing healthcare and transforming economics
of the Rx business. Will need to craft ways of dealing withindustrys new economic landscape.
A single herd of goats maysoon replace a $150 milliondrug factoryHBR 4/2000
2006: ATryn approved for DVT
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The Human Genome & Biotechnology
A milestone in biology unlike any other.
Weve called the human genome the book of life, butits really 3 books: Its a history book. Its a shopmanual and parts list. And, its a textbook of medicinemore profoundly detailed than ever.
--Francis Collins, former director NHRI director
A short 50 years after the discovery of DNA by Watsonand Crick in 1953
A complete list of coded instructions to make a person
Would fill a stack of paperbacks 200 feet high
50 years to type at 8 hours/day, 60 wpm
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The Human Genome: Fun Facts
30,000-40,000 genes not the 100-120,000 thought earlier Five times as many as in bakers yeast
About twice as many as that needed to grow a worm orfly!
Bananas share about our genome whilemice share 90%!
BUT, each single human gene can make 10 proteins vs. aworm or flys genes making just one or two.
We have the Cuisinart vs. the paring knife--Francis Collins
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5 million strands of DNA can fit through the eye of a
needle
All our DNA laid end to end would go to the sun and back600 times!
The genetic instructions for making a person take up lessthan 1 of the 6-ft long strand of DNA in each cell
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Since HGP
ENCODE: the Encyclopedia OfDNA Elements, in 9/2003, to IDall functional elements in thehuman genome sequence
HAPMAP: haplotype map, will
allow researchers to findgenes/genetic variations thataffect health and disease: 100IDd so far
1000 Genomes: extends HAPMAP
through global collaboration tomap 1,000 genomes in 3 years
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Since HPG
NIH Roadmap Project on genotype tissueexpression: are variants of disease riskassociated in relevant tissue
Knock-Out Mouse Project (KOMP)
Mammalian gene collection Cancer Genome Atlas: feasibility of full-scale
effort to systematically explore entire spectrumof genomic changes involved in human cancer
Molecular manual of disease created: 12/2008 Proteinpedia being created: largest free
resource of experimental info human proteins
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Goal
Diagnostics to prevention
Pharmacogenomic knowledge transfer
to therapeutics for gene therapy, drugtherapy
Personalize genome + familyhistory=personalized medicine
Other non-profit commercial effortsunderway
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Other Efforts
Bringing the genome into the light, Church says, isthe great project of our day.
To Church, who built his first computer at age 9 and
taught himself three programming languages by 15,all of this is unfolding according to the same laws ofexponential progress that have propelled digitaltechnologies, from computer memory to the Internetitself, over the past 40 years: Moore's law for circuits
and Metcalfe's law for networks. These principles arenow at play in genetics, he argues, particularly inDNA sequencing and DNA synthesis.
---Wired
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Personal Genome Mapping
Polonator G.007 $150K machine usingopen architecture like IBM in 80s
fueling PC revolution DeCodeMe
23andMe
Navigenics KNOME
Price range $400 to $350,000
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Gene Map Becomes a Luxury Item 3/2008 Id rather spend my money on my genome than a
Bentley or an airplane. Dan Stoicescu, millionaire retired Biotech exec
X Prize Foundation: $10 million to first group tosequence 100 human genomes in 10 days for $10Kor less
Commercial goal: get price to $1K or less Has dropped by 4 orders of magnitude in 5 years
Scientists call for better government regulation ofgenetic tests
DNA databases blocked from public by NIH afterstudy show a new type of DNA analysis could confirmID of individual in a pool of masked data if that
persons gene profile was already known
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Future Environment
Personalization of medicine
Consumer empowermentpreventivecare
Consumer ignoranceacute care
MultibusterspharmacogeneticsBlockbustersUniversal Rx
Patient variabilitytailored carePatient uniformitycare standards
Disease heterogeneitycustomization, targeted Rx
Disease uniformityguidelines,formularies
Disease by mechanicsprognosisDisease by symptomsdiagnosis
FuturePresent
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Personalized Medicine Poised for
Progress in 2009 12/24/2008
poised for dramatic progress in 2009
in the clinic and laboratory personal, predictive, preventive
Weve finally gotten to the point where
we can tell people about how their DNAimpacts their health, Elissa Levin,23andMe
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Promises and Implications BIG*
Promise: Rapid technological innovation and vast number of new targets
identified and predictive tests
Today 483 targets account for all Rx drugs marketed, tomorrow 5-10,000 targets
Rapid acceleration in pace of new therapeutic introductions
Drugs and treatments that are more tailored to specific patients
Implications:
Development becomes a bottleneck
Shorter product life cycles Market fragmentation
Blurring distinction between product and service* Gary Pisano, HBS 3/2000, adapted
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Specialty Pharmaceuticals
Created when many products moved frommedical side to the pharmacy side of thebudget=PBM control
No specific criteria, but general attributes: Expense with annual therapy costs between
$20,000 and $250,000
Treatment for chronic condition, possible lifetime
therapy Special handling, route of administration, patient
support
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What Does It Mean to the Market?
IMS Health estimatesspecialty pharmacy sales of$85 billion in 2008
Expected to reach $100
billion in 2010 18% growth next 2 years
Global biotech Rx sales grew12.5% 2007 to $75 billion
Biotech growing 2x pharma(6.4%)
US sales 56% of total ($42
billion)
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Market Trends
22 biotech products generated>$1 billion sales compared to 6in 2002
Biotech products represent25% of the Rx pipeline
http://www.phrma.org/files/Biotech%202008.pdf
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Biotech Product Trends
Development of biotech compounds isexplosive
633 biotech medicines in US pipeline
2001: Most compounds focused in theoncology arena, followed by infectiousdisease, CV and neuroscience
2007: Cancer 254, infectious disease 162,autoimmune disease 59, HIV/AIDS 34, CV
25, diabetes 19 Breakthrough treatments may provide
new hope of some diseases
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Major Diseases
Autoimmune: rheumatoid arthritis, MS,lupus, chronic fatigue syndrome
CV: CHF, MI, PAD, hypercholesterimia Gene disorders: CF, Gauchers
ID: Hep A, B, C, tuberculosis, anthrax,
bird flu Neurodisorders: AD, PD, muscular
dystrophy, spinal cord injuries
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Impact on People
Cancer 1.5 million US $219 B
CVD 71 million US $403 B
Alzheimers 5 million US $148 B Parkinsons 1 million US $35.5 B
MS 400,000 US $10 B
Diabetes 24 million US $132 B HIV 1.2 million US $37 B
Hep A B C >5 million US $3 B
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Therapeutic Technologies
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Pharming
FDA guidelinesgoverning geneticengineering ofanimals for food,drugs, or medicaldevices
Released 9/2008
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. . . Contact us for the rest of
the presentation
www.catalystenterprises.net
[email protected] Tel: 651-905-9002
http://www.catalystenterprises.net/http://www.catalystenterprises.net/