1.1 What Is Biotechnology and What Does It Mean to You? (1 of 13) Biotechnology —broadly defined as using living organisms, or the products of living organisms, for human benefit (or to benefit human surroundings) to make a product or solve a problem: Remember this definition! Historical Examples: Early ancestors also took advantage of microorganism Fermentation (anaerobic) Selective breeding Use of antibiotics
1.1 What Is Biotechnology and What Does It Mean to You? (2 of 13) Examples of Biotechnology are in Your Home ( a) Kitchen biotechnology includes breads, cheeses, yogurts, and many other foods and drinks. ( b) A smartphones that monitor vital signs and blood chemistry such as blood sugar levels that can detect blood glucose levels in a test strip.
1.1 What Is Biotechnology and What Does It Mean to You? (3 of 13) Example of Biotechnology – Selective Breeding Figure 1.2 Selective Breeding Is an Old Example of Biotechnology That Is Still Common Today (a) Corn grown by selective breeding. ( b ) Zebrafish an important experimental model organism
1.1 What Is Biotechnology and What Does It Mean to You? (4 of 13) Modern Examples: Use of antibiotics: Batch (large-scale) processes Gene cloning Genetic engineering Recombinant D N A technology Human Genome Project: Genomics (the study of genomes) Artificial or synthetic genomes Genome editing: C R I S P R- Cas
1.1 What Is Biotechnology and What Does It Mean to You? (5 of 13) Figure 1.3 Gene Maps of Chromosomes 13 and 21. The Human Genome Project has led to the identification of nearly all human genes and has mapped their location on each chromosome . The figure display partial lists of genes known to be involved in human genetic diseases. Now use the link to further study the diseases involved in these chromosomes http://ghr.nlm.nih.gov/chromosome
1.1 What Is Biotechnology and What Does It Mean to You? (6 of 13) Example of “Modern” Biotechnology : Recombinant D N A technology started modern biotech as an industry Examples of applications: Development of disease-resistant plants Food crops that produce greater yields “Golden rice” engineered to be more nutritious Genetically engineered bacteria that can degrade environmental pollutants Work in groups to come up with more examples of applications
1.1 What Is Biotechnology and What Does It Mean to You? (7 of 13) The Do-It-Yourself (DIY) Biotechnology Movement: D I Y biotechnology: Individuals with many different backgrounds with an interest in tinkering, to entrepreneurs. They work in communal space. Some have referred to DIY participants as “ biohackers”. No government funding for D I Y biotechnology (work in unregulated environment). Biotechnology: A Science of Many Disciplines: Piecing together complex information from different scientific disciplines
1.1 What Is Biotechnology and What Does It Mean to You? (8 of 13) Based on this tree, studying biology is not enough to become successful in the biotech industry? Figure 1.4 The Biotechnology Tree: Different Disciplines Contribute to Biotechnology. Notice that the “roots” are primarily formed by work in the basic sciences —research into fundamental processes of living organisms at the biochemical, molecular , and genetic levels.
1.1 What Is Biotechnology and What Does It Mean to You? (9 of 13) Products of Modern Biotechnology: Example of proteins created by gene cloning called recombinant proteins Table 1.2 Examples of Recombinant Proteins Manufactured from Cloned Genes Product Application Blood Factor VIII (clotting factor) Treat haemophilia Epidermal growth factor Stimulate antibody production in patients with immune system disorders Growth hormone Correct pituitary deficiencies and short stature in humans; other forms are used in cows to increase milk production Insulin * *** Treat diabetes Interferons Treat cancer and viral infections Interleukins Treat cancer and stimulate antibody production Monoclonal antibodies Diagnose and treat a variety of diseases including arthritis and cancer Tissue plasminogen activator Treat heart attacks and stroke
1.1 What Is Biotechnology and What Does It Mean to You? (10 of 13) Brief list of some of the top-selling biotechnology drugs and the companies that developed them: Table 1.1 *2016—Top 10 Biotechnology Drugs (Each with Worldwide Sales over $5 Billion) Drug Name Developer Drug Type Function (Treatment of Human Disease Conditions) Humira AbbVie Antibody (monoclonal) Rheumatoid arthritis, Crohn’s disease, Ulcerative colitis Harvoni Gilead Sciences Small molecule Hepatitis C Rituxan Roche Antibody (monoclonal) Non-Hodgkin’s lymphoma Revlimid Celgene Small molecule Multiple myeloma Avastin Roche Antibody (monoclonal) Colorectal cancer; breast cancer; non–small cell lung cancer; ovarian, brain, and cervical cancer Herceptin Roche Antibody (monoclonal) Breast cancer, gastric cancer Enbrel Amgen Recombinant protein Rheumatoid arthritis, psoriasis Prevnar 13 Pfizer Vaccine Pneumococcal ( Streptococcus Pneumoniae ) antibacterial vaccine Lantus Sanofi Peptide Diabetes mellitus types I and II Neulasta Amgen Recombinant protein Anemia (neutropenia/leukopenia) *Data based on the most recent source available at the time of publication: Morrison C, Lähteenmäki R. Public biotech in 2016—the numbers. Nat Biotechnol. 2017;35:623–629.
1.1 What Is Biotechnology and What Does It Mean to You? (11 of 13) Most drugs are developed to combat diseases affecting humans—Why? Figure 1.6 Investigational Biotechnology Drugs by Disease Category
1.1 What Is Biotechnology and What Does It Mean to You? (12 of 13) Use genetically modified cultured cells to make protein of interest Genes of interest can be introduced into bacterial or mammalian cells. Such cells can be grown using cell culture techniques. Recombinant proteins isolated from these cells are isolated.
1.1 What Is Biotechnology and What Does It Mean to You? (13 of 13) Gene Therapy Approaches: Attempts to treat and cure human disease conditions: Delivering genes to treat or cure a genetic disorder: Grow organs for transplantation New biotechnology products from marine organisms are being used to treat cancers, strokes, and arthritis Ethics and Biotechnology Figure 1.7 Biotechnology Is a Controversial Science That Presents Many Ethical Dilemmas GMO: Genetically Modified Organism
1.2 Types of Biotechnology (1 of 24) APPLICATIONS Microbial Biotechnology Agricultural Biotechnology Animal Biotechnology Forensic Biotechnology Bioremediation Aquatic Biotechnology Medical Biotechnology Biotechnology Regulations
1.2 Types of Biotechnology (2 of 24) Microbial Biotechnology —manipulation of microorganisms such as yeast and bacteria: Create better enzymes More efficient decontamination processes for industrial waste product removal Used to clone and produce large amounts of important proteins used in human medicine Creation of synthetic genomes: Manmade D N A sequences that can be used to engineer microbes with desirable characteristics
1.2 Types of Biotechnology (3 of 24) Agricultural Biotechnology: United Nations Food and Agricultural Organization predicts by 2050, we will need to feed a world population of 9.1 billion! This requires raising food production by approximately 70 percent! Gene-edited crops: C R I S P R-Cas editing tools Work in groups to brainstorm a few solutions to better feed the world by 2050
1.2 Types of Biotechnology (4 of 24) The Common White Button Mushroom is the First Gene-Edited Crop Created by C R I S P R to be Approved for Human Consumption. (Using CRISPR- Cas editing tools and not genetically modified)
1.2 Types of Biotechnology (5 of 24) Agricultural Biotechnology: Plants are more environmentally friendly that yield more per acre (genetically engineered) Resistance to diseases and insects Foods with higher protein or vitamin content Drugs developed and grown as plant products These better plants ultimately reduce production costs to help feed the growing world population
1.2 Types of Biotechnology (6 of 24) Agricultural Biotechnology: Work in groups to discuss how you can use this technology in a third world country to create a better corn crop (main crop in that country) that contains all of the 22 essential amino acids (improve nutrient content). Seed storage proteins. Will improve crops that are created to satisfy world hunger reduce available land for biofuel crops? Discuss in groups
1.2 Types of Biotechnology (8 of 24) Animal Biotechnology: Animals as a source of medically valuable proteins: Antibodies Transgenic animals Animals as important models in basic research: Gene “knockout” experiments Design and testing of drugs and genetic therapies Animal cloning: Source of transplant organs
1.2 Types of Biotechnology (9 of 24) Hornless Cattle without Pain Target the horn-growing genes using “ molecular scissors.” This means that scientists sliced out the horn-growing genes naturally found in Holsteins and replaced them with genes that stop horns from growing in the Angus breed
1.2 Types of Biotechnology (10 of 24) Cultured cells produce burger in a dish Cultured beef muscle cells have been used to create meat with varied colors and incorporated fat cells to improve taste .
1.2 Types of Biotechnology (11 of 24) Animal Biotechnology: Transgenic animal —way to achieve large-scale production of therapeutic proteins from animals for use in humans Female transgenic animals express therapeutic proteins in milk (contains genes from another source) Example: Human genes coding for clotting proteins can be introduced into female goats for production of these proteins in their milk
1.2 Types of Biotechnology (12 of 24) Animal Biotechnology: Gene knockout: Disrupt a gene in the animal and then look at what functions are affected in the animal as a result of the loss of the gene This allows researchers to determine the role and function of the gene Since humans are similar to rats and mice, gene knockout studies in rats and mice can lead to better understanding of gene function in humans. Work in groups and give an example of a gene you would like to knockout in mice.
1.2 Types of Biotechnology (13 of 24) Forensic Biotechnology: D N A fingerprinting: Inclusion or exclusion of a person from suspicion Paternity cases Identification of human remains Endangered species Tracking and confirmation of the spread of disease Liquid biopsies for cancer detection and monitoring are becoming a big area of forensic interest Guardant Health, Exosome Diagnostics, and Illumina have invested millions in this blood-based analysis process
1.2 Types of Biotechnology (14 of 24) D N A detection of markers for disease has expanded D N A forensics NO SIGNAL SIGNAL EXAMPLE: Three genes used as indicators of certain types of bladder cancer. Detection in urine can be obtained with 90.9 percent accuracy. W hen tumor DNA is present , it combines with bound complementary DNA detectors to release a signal.
1.2 Types of Biotechnology (15 of 24) Bioremediation: The use of biotechnology to process and degrade a variety of natural and manmade substances: Particularly those that contribute to environmental pollution Example—stimulated growth of bacteria that degrade components in crude oil: 1989 Exxon Valdez oil spill in Alaska 2010 Deep Water Horizon spill promoted research into natural oil-degrading organisms and enzymes
1.2 Types of Biotechnology (16 of 24) Bioremediation — adding new bacteria and/or nutrients to stimulate growth of bacteria to clean up oil spill Figure 1.12 Bioremediation in Action Strains of the bacterium Pseudomonas were used to help clean Alaskan beaches following the Exxon Valdez oil spill. Scientists on this Alaskan beach are applying nutrients that will stimulate the growth of Pseudomonas to help speed up the bioremediation process .
1.2 Types of Biotechnology (17 of 24) Aquatic Biotechnology: Aquaculture —raising finfish or shellfish in controlled conditions for use as food sources: 50% of all fish consumed by humans worldwide Use of genetic engineering to produce disease-resistant strains of oysters and vaccines against viruses that infect salmon and other finfish: 2015 – F D A approved Aqu Advantage® salmon as the first G M animal for human consumption Transgenic salmon overproduce growth hormone, leading to extraordinary growth rates over short growing periods and thus decreasing the time and expense required to grow salmon for market sale
1.2 Types of Biotechnology (18 of 24) Aquatic Biotechnology Why create transgenic salmon overproducing growth hormone? Figure 1.13 Genetically Engineered Salmon Became the First G M Animals Approved for Human Consumption Transgenic Normal How does this modified salmon help humans? Genetically modified salmon (top) bred to grow to adult size for market sale in half the time of a non-GM salmon (Normal or Wild type ).
1.2 Types of Biotechnology (19 of 24) Aquatic Biotechnology: Genetic engineering: Disease-resistant strains of oysters Vaccines against viruses that infect salmon and other finfish Transgenic salmon that overproduces growth hormone Bioprospecting: Rich and valuable sources of new genes, proteins, and metabolic processes with important applications for human benefits: Marine plankton and snails found to be rich sources of antitumor and anticancer molecules
1.2 Types of Biotechnology (20 of 24) Medical Biotechnology: Involved with the whole spectrum of human medicine: Preventive medicine Diagnosis of health and illness Treatment of human diseases New information from Human Genome Project: Gene therapy: C R I S P R-Cas—technique for genome editing that provides molecular scissors of sorts to cut out and replace a specific sequence of D N A—has potential to treat diseases Immunotherapy Stem cell technologies
1.2 Types of Biotechnology (21 of 24) Stem cells —grown in lab and then treated with different chemicals to allow them to develop into specific kinds of tissues needed for transplant Current use: Stem cells are used for diabetes; spinal cord injuries Work in groups to come up with a list of other diseases you have read about in the newspaper or heard on the news that scientists are testing with stem cells
1.2 Types of Biotechnology (22 of 24) Medical Biotechnology: Genes and Genomes are headline news items
1.2 Types of Biotechnology (23 of 24) Biotechnology Regulations: Essential aspect of the biotechnology business involves the regulatory processes that govern the industry Most biotechnology products must are carefully examined before they are available for use 2015 – F D A accepted a new drug application (N D A) for the first sensor-embedded version of Abilify for depression: Monitoring device for behavioral health Otsuka Pharmaceuticals and Proteus Digital Health Why as a consumer should you care about a product undergoing intense regulations?
1.2 Types of Biotechnology (24 of 24) The Biotechnology “Big Picture” Important to remember that almost all areas of biotechnology are closely interrelated: Examples: Applications of bioremediation are heavily based on using microbes (microbial biotechnology) to clean up environmental conditions Medical biotechnology relies on the use of microbes to produce recombinant proteins Industrial biotechnology—application of biotechnology to industrial processes such as manufacturing
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (1 of 11) How will medical biotechnology change our lives in the years ahead? Human Genome Project: Research on the function of human genes and controlling factors that regulate genes Human proteome: Collection of proteins responsible for activity in a human cell
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (2 of 11) How will medical biotechnology change our lives in the years ahead? Personal genomics: Precision medicine Pharmacogenomics Single Nucleotide Polymorphisms (S N Ps): Single nucleotide changes (mutations) in D N A sequences that vary from individual to individual These variations are the cause of some genetic diseases (sickle cell anemia) S N Ps will help identify genes involved in medical conditions including arthritis, stroke, cancer, heart disease, diabetes, and behavioral and emotional illnesses
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (3 of 11) Example of S N Ps and breast cancer Identification of S N Ps in B R C A1 and B R C A2 genes involved in promoting breast cancer led to development of better targeted treatments for people who have those specific gene mutations Can you think of how this knowledge might be useful for someone who is not already diagnosed with cancer? (Hint: Think of your basic knowledge of genetics.)
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (4 of 11) Example of how we can benefit from the human genome project Figure 1.15 Single-Nucleotide Polymorphisms Based on the figure, why doesn’t person 2 develop a genetic disease due to the S N P (G → T)?
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (5 of 11) How can you test one person’s D N A for many different S N Ps? Microarray (gene chip): Isolate D N A from patient Apply this sample to a microarray which contains many D N A sequences Compare patterns of D N A binding between patient’s D N A and D N A on microarray to reveal patient’s S N P patterns
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (6 of 11) How will medical biotechnology change our lives in the years ahead? Pharmacogenomics is customized medicine: Tailor-designing drug therapy and treatment strategies based on the genetic profile of a patient: Do microarray analysis and then design drugs against genes that are mutated for an individual patient Metabolomics: A snapshot of the small molecules produced during cellular metabolism: Glucose, cholesterol, A T P, and signaling molecules Can distinguish between disease process and physiological adaptation
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (7 of 11) How will medical biotechnology change our lives in the years ahead? Nanotechnology: Applications that incorporate extremely small devices Small particles that can deliver drugs (also genes, RNA, antibodies …) to cells Figure 1.16 Nanotechnology in Action Nanoparticles can be directed to target disease cells. In this way, drugs that cannot pass through the cell membrane can be released inside these target cancer cells.
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (8 of 11) Gene Therapy Technology: Replacing or augmenting defective genes with normal copies of the gene: Still have barriers to overcome before this technology becomes safe and effective: Obstacles include the following: How can normal genes be delivered to virtually all cells in the body? What are the long-term effects of introducing extra genes in humans? What must be done to ensure the proper protein is made after the genes are delivered to the body?
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (9 of 11) Stem Cell Technology: Stem cells are immature cells that grow and divide to produce different cell types Most stem cells are from embryos called embryonic stem cells (E S Cs) but they are controversial since the process involves death of an embryo Some stem cells are from adult cells (A S Cs): Either type of stem cell can be coaxed to grow into cells of interest to replace damaged tissue or failing organs (liver, pancreas, and retina) Work in groups to explain why scientists are doing more research using embryonic versus adult stem cells
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (10 of 11) Embryonic stem cells can give rise to many types of differentiated cells
1.3 What Will the New Biotechnology Century Look Like? an Example from Medical Biotechnology (11 of 11) How will medical biotechnology change our lives in the years ahead? Regenerative medicine: Genetically modifying stem cells of patients to treat genetic disease conditions In future, scientists will be able to: Isolate adult stem cells from a patient with a genetic disorder Genetically manipulate these cells by gene therapy approaches Reinsert the cells into the same patient to help treat their genetic disease
1.4 The Biotechnology Workforce (1 of 8) The Business of Biotechnology: Today, biotech companies generate global revenues of $163 billion. This makes up about 20 percent of the revenues generated by the pharmaceutical market. Annual sales of biotherapeutics are in excess of $225 billion worldwide and doubling about every five years!
1.4 The Biotechnology Workforce (2 of 8) Top Regions for Biotechnology Jobs Figure 1.19 Biotech Companies Around the World For the names of the companies https ://www.biotech-careers.org /
1.4 The Biotechnology Workforce (3 of 8) What Is a Biotechnology Company? What is the difference between a biotechnology company and a pharmaceutical company? Pharmaceutical companies are involved in drug development (chemically synthesizing or purifying compounds to make the drug) Pharmaceutical company does not use living organisms to grow or produce a product Biotech company is involved in drug development using live organisms Biotech companies also create products that are not drugs
1.4 The Biotechnology Workforce (4 of 8) Table 1.3 Top Five Biotechnology Companies and Top Five Pharmaceutical Companies by Revenue in 2017 Biotech Companies Revenue (Billions) Amgen $129 Gilead Sciences $103 Novo Nordisk $ 96 Celgene $ 77 Biogen $ 66 Pharma Companies Revenue (Billions) Johnson & Johnson $379 Novartis $216 Pfizer $208 Roche $199 AbbVie $153 Adapted from Genetic Engineering & Biotechnology News, https://www.genengnews.com/the-lists/top-25-biotechcompanies-of-2017/77901002 and https://www.genengnews.com/the-lists/top-10-pharma-companies-of-2017/77901005 . Revenue based on preliminary results reported by companies.
1.4 The Biotechnology Workforce (5 of 8) How does a biotech company start? Generally created as a start-up company with few employees backed by venture capital investments (derived from financial institutions and private donors called angel investors) Why invest as an angel donor? To ultimately have company ownership in exchange for donating funds – get rich if company is successful! Takes 10 years to get a product to market at a big cost! Eventually start-ups are bought out by bigger established companies Bringing drug close to market = value to the company File for initial public offering (I P O) stock
1.4 The Biotechnology Workforce (6 of 8) Jobs in Biotechnology: Research and development: Preclinical, discovery research, bioinformatics, and lab safety Operations: Process/product development, manufacturing, and production Quality: Quality control and assurance Clinical research: Clinical research and regulatory affairs Finance and administration: Finance, business development, administration, information systems, legal, and facilities management
1.4 The Biotechnology Workforce (7 of 8) Organizational structure of medium-sized biotech company
1.4 The Biotechnology Workforce (8 of 8) What is the best approach to landing a good job in a biotech company? Obtain a degree in molecular biology, biochemistry, or bioinformatics Minor in information technology Take lots of math courses: Allows for unique combinations Have great skills in oral and written communication Must be a collaborative team player Why go into this field? It pays well and is personally rewarding!