Bio- and Nanotechnology: Emerging Medical Miracles on the Horizon

Marsha K. Millonig, MBA, RPh President & CEO

Catalyst Enterprises, LCC

Today’s Objectives

• Understand biotechnology and nanotechnology• Describe the biotechnology market size• Explain what fields are parts of the emerging

business 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

Why is it Important?

Global Population Growth• Earth’s capacity is estimated at 12 billion people• It took 100 million years before Earth had 1 billion

people in 1830• Yet only 170 years for the population to reach 6

billion plus• 2005: 6,436,562,930 • 2009: 6,752,560,109• ½ the people who have ever lived are on the planet

today• Less than 100 years before we reach capacity

Biotechnology is needed to…

• Create better fuels that don’t harm environment

• Create tools to clean environment, feed a burgeoning global population, cure untold human suffering

Biotechnology Definition• Use of cellular and biomolecular processes to

solve problems or make useful products. Life sciences…biology/chemistry technology affecting discovery and development of products for:

–Healthcare (therapeutics, diagnostics, drug delivery, cell and gene 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……

Development is evolutionary…

4000-2000 BC: biotech used to leaven bread & ferment beer

1830: Proteins discovered1833: First enzyme

discovered1865: Genetic science

begins-Mendel discovers laws of heredity

1879: Chromosomes discovered

Development is evolutionary…

1906: The term genetics introduced1919: The word biotechnology is first

used in print1938: The term molecular biology

coined1941: The term genetic engineering

is first used

Development is evolutionary…

1953: Watson and Crick DNA Structure

1958: DNA made in test tubeSickle 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 a specific site

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 produced1980: Oil-eating microbes patented by Exxon1982: First recombinant DNA vaccine for

livestock1983: First whole plant grown from

biotechnology

Development is evolutionary…

1980: First gene-synthesizers developed

1981: First transgenic animals1982: First biotech drug:

insulin1983: First artificial

chromosome synthesizedFirst genetic markers for

inherited disease found

Development is evolutionary…

1984: DNA fingerprinting developed1985: Genetic fingerprinting

entered as evidence in courtroom1986: Interferon first anti-cancer

drug from biotechFirst genetically engineered vaccine

for humans: Hepatitis BMicrobes used to clean oil spill

…but speeding up.1988: First US patent for genetically altered

animal—a transgenic mouse1989: First DNA exoneration now 216 (4/08)1990: First food product from biotech

approved: modified yeast1994: First FDA approval for first whole food

product: FLAVRSAVR™ tomato1997: First weed & insect resistant crops

developedFirst cloned animal: Hello Dolly!

…but speeding up.

1998: Human embryonic stem cells lines established

Herceptin approved-considered first pharmacogenomic (personalized) medicine

First complete animal genome: roundworm2000: First complete map of a plant genomeFirst draft human genome

…but speeding up. 2004: First genetically modified pet: the

GloFishFDA clears genotyping test to aid in

medication selectionFirst cloned pet, a kitten2005: Skin cells converted to embryonic

stem cells1 billionth acre biotech seed plantedFirst complete dog genome: boxer2006: FDA approves Gardasil-first vaccine

for cancer-causing virus

…but speeding up. 2007: Successful reprogramming human skin cells to

create cells indistinguishable from embryonic stem cells

Biotech cattle that cannot develop prions=no mad cow disease

2008: draft corn genome10 disease bearing stem cell lines createdMature human embryos created from adult skin cells:

personalized stem cells for disease treatmentFirst synthetic genome: the road to creating lifeFirst complete map of cancer patient genomeFirst US FDA application for gene-therapy/cancer

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 communication companies, 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 with industry’s new economic landscape.

A single herd of goats may soon replace a $150 million drug factory…HBR 4/2000

2006: ATryn approved for DVT

The Human Genome & Biotechnology

• “A milestone in biology unlike any other.”• “We’ve called the human genome the book of life, but it’s really

3 books: It’s a history book. It’s a shop manual and parts list. And, it’s a textbook of medicine more profoundly detailed than ever.”

» --Francis Collins, former director NHRI director

• A short 50 years after the discovery of DNA by Watson and 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

The Human Genome: Fun Facts

• 30,000-40,000 genes not the 100-120,000 thought earlier• Five times as many as in baker’s yeast• About twice as many as that needed to grow a worm or fly!• Bananas share about ½ our genome while mice share 90%!

• BUT, each single human gene can make 10 proteins vs. a worm or fly’s genes making just one or two.

• “We have the Cuisinart vs. the paring knife--Francis Collins

• 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 back 600 times!

• The genetic instructions for making a person take up less than 1” of the 6-ft long strand of DNA in each cell

Since HGP…

• ENCODE: the Encyclopedia Of DNA Elements, in 9/2003, to ID all functional elements in the human genome sequence

• HAPMAP: haplotype map, will allow researchers to find genes/genetic variations that affect health and disease: 100 ID’d so far

• 1000 Genomes: extends HAPMAP through global collaboration to map 1,000 genomes in 3 years

Since HPG• NIH Roadmap Project on genotype tissue

expression: are variants of disease risk associated in relevant tissue

• Knock-Out Mouse Project (KOMP)• Mammalian gene collection• Cancer Genome Atlas: feasibility of full-scale

effort to systematically explore entire spectrum of genomic changes involved in human cancer

• Molecular manual of disease created: 12/2008• Proteinpedia being created: largest free resource

of experimental info human proteins

Goal

• Diagnostics to prevention• Pharmacogenomic knowledge

transfer to therapeutics for gene therapy, drug therapy

• Personalize genome + family history=personalized medicine

• Other non-profit commercial efforts underway

Other Efforts

• Bringing the genome into the light, Church says, is the 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 of exponential progress that have propelled digital technologies, from computer memory to the Internet itself, over the past 40 years: Moore's law for circuits and Metcalfe's law for networks. These principles are now at play in genetics, he argues, particularly in DNA sequencing and DNA synthesis.”

» ---Wired

Personal Genome Mapping

• Polonator G.007 $150K machine using open architecture like IBM in 80’s fueling PC revolution

• DeCodeMe • 23andMe• Navigenics• KNOME• Price range $400 to $350,000

Gene Map Becomes a Luxury Item…• 3/2008

• “I’d 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 to sequence 100 human genomes in 10 days for $10K or 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 of

genetic tests• DNA databases blocked from public by NIH after study

show a new type of DNA analysis could confirm ID of individual in a pool of masked data if that person’s gene profile was already known

Future Environment

Personalization of medicine

Consumer empowerment–preventive care

Consumer ignorance–acute care

Multibusters–pharmacogeneticsBlockbusters–Universal Rx

Patient variability–tailored carePatient uniformity–care standards

Disease heterogeneity–customization, targeted Rx

Disease uniformity–guidelines, formularies

Disease by mechanics–prognosis

Disease by symptoms–diagnosis

FuturePresent

“Personalized Medicine Poised for Progress in 2009”

• 12/24/2008• “poised for dramatic progress in

2009” in the clinic and laboratory• “personal, predictive, preventive”• “We’ve finally gotten to the point

where we can tell people about how their DNA impacts their health,” Elissa Levin, 23andMe

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

Specialty Pharmaceuticals

• Created when many products moved from “medical” side to the “pharmacy” side of the budget=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

What Does It Mean to the Market?

• IMS Health estimates specialty pharmacy sales of $85 billion in 2008

• Expected to reach $100 billion in 2010

• 18% growth next 2 years• Global biotech Rx sales grew

12.5% 2007 to $75 billion• Biotech growing 2x pharma

(6.4%)• US sales 56% of total ($42

billion)

Market Trends

• 22 biotech products generated >$1 billion sales compared to 6 in 2002

• Biotech products represent 25% of the Rx pipeline

• http://www.phrma.org/files/Biotech%202008.pdf

Biotech Product Trends• Development of biotech compounds is

explosive• 633 biotech medicines in US pipeline • 2001: Most compounds focused in the

oncology arena, followed by infectious disease, 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

Major Diseases

• Autoimmune: rheumatoid arthritis, MS, lupus, chronic fatigue syndrome

• CV: CHF, MI, PAD, hypercholesterimia• Gene disorders: CF, Gaucher’s• ID: Hep A, B, C, tuberculosis,

anthrax, bird flu• Neurodisorders: AD, PD, muscular

dystrophy, spinal cord injuries

Impact on People

• Cancer 1.5 million US $219 B• CVD 71 million US $403 B• Alzheimer’s 5 million US $148 B• Parkinson’s 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

Therapeutic Technologies

Pharming

• FDA guidelines governing genetic engineering of animals for food, drugs, or medical devices

• Released 9/2008

. . . Contact us for the rest of the presentation

• www.catalystenterprises.net• mmillonig@catalystenterprise

s.net• Tel: 651-905-9002