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Bio- and Nanotechnology: Emerging Medical Miracles on the Horizon

Bio- and Nanotechnology: Emerging Medical Miracles on the Horizon. Marsha K. Millonig, MBA, RPh President & CEO Catalyst Enterprises, LCC. Today’s Objectives.

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Bio- and Nanotechnology: Emerging Medical Miracles on the Horizon

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  1. Bio- and Nanotechnology: Emerging Medical Miracles on the Horizon Marsha K. Millonig, MBA, RPh President & CEO Catalyst Enterprises, LCC

  2. 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

  3. Why is it Important?

  4. 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

  5. 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

  6. 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……

  7. Development is evolutionary… 4000-2000 BC: biotech used to leaven bread & ferment beer 1830: Proteins discovered 1833: First enzyme discovered 1865: Genetic science begins-Mendel discovers laws of heredity 1879: Chromosomes discovered

  8. 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 is first used

  9. Development is evolutionary… 1953: Watson and Crick DNA Structure 1958: DNA made in test tube Sickle cell caused by AA change 1960: Messenger RNA discovered 1967: First automatic protein sequencer is perfected 1969: Enzyme synthesized in vitro the first time 1970: First enzyme discovered to cut DNA molecules at a specific site

  10. Development is evolutionary… 1971: First complete synthesis of a gene 1973: First time DNA fragments linked 1975: First monoclonal antibodies made 1976: First NIH research guidelines Boyer co-founds Genentech, 1st bio co. 1978: Recombinant insulin first produced 1980: Oil-eating microbes patented by Exxon 1982: First recombinant DNA vaccine for livestock 1983: First whole plant grown from biotechnology

  11. Development is evolutionary… 1980: First gene-synthesizers developed 1981: First transgenic animals 1982: First biotech drug: insulin 1983: First artificial chromosome synthesized First genetic markers for inherited disease found

  12. Development is evolutionary… 1984: DNA fingerprinting developed 1985: Genetic fingerprinting entered as evidence in courtroom 1986: Interferon first anti-cancer drug from biotech First genetically engineered vaccine for humans: Hepatitis B Microbes used to clean oil spill

  13. …but speeding up. 1988: First US patent for genetically altered animal—a transgenic mouse 1989: First DNA exoneration now 216 (4/08) 1990: First food product from biotech approved: modified yeast 1994: First FDA approval for first whole food product: FLAVRSAVR™ tomato 1997: First weed & insect resistant crops developed First cloned animal: Hello Dolly!

  14. …but speeding up. 1998: Human embryonic stem cells lines established Herceptin approved-considered first pharmacogenomic (personalized) medicine First complete animal genome: roundworm 2000: First complete map of a plant genome First draft human genome

  15. …but speeding up. 2004: First genetically modified pet: the GloFish FDA clears genotyping test to aid in medication selection First cloned pet, a kitten 2005: Skin cells converted to embryonic stem cells 1 billionth acre biotech seed planted First complete dog genome: boxer 2006: FDA approves Gardasil-first vaccine for cancer-causing virus

  16. …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 genome 10 disease bearing stem cell lines created Mature human embryos created from adult skin cells: personalized stem cells for disease treatment First synthetic genome: the road to creating life First complete map of cancer patient genome First US FDA application for gene-therapy/cancer

  17. 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 2005 and Today

  18. 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

  19. 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

  20. 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

  21. 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

  22. 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

  23. 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

  24. 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

  25. 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

  26. 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

  27. Present Future Disease by symptoms–diagnosis Disease by mechanics–prognosis Disease uniformity–guidelines, formularies Disease heterogeneity–customization, targeted Rx Patient uniformity–care standards Patient variability–tailored care Blockbusters–Universal Rx Multibusters–pharmacogenetics Consumer ignorance–acute care Consumer empowerment–preventive care Future Environment Personalization of medicine

  28. “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

  29. 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

  30. 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

  31. 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)

  32. 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

  33. 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

  34. 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

  35. 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

  36. Therapeutic Technologies

  37. FDA guidelines governing genetic engineering of animals for food, drugs, or medical devices Released 9/2008 Pharming

  38. . . . Contact us for the rest of the presentation • www.catalystenterprises.net • mmillonig@catalystenterprises.net • Tel: 651-905-9002

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