Millon i g Asap 12009 Partial

7/28/2019 Millon i g Asap 12009 Partial

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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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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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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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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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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=1


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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=1


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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=1


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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/

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