Pharmaceutical Biotechnology Lecture 1 PDF

Pharmaceutical Biotechnology Introduction: Principles and Applications Lecture 1 Assistant Prof. Dr. Mohammed Talib Ph.D. in Biotechnology College of Pharmacy the University of Misan What is biotechnology ? Biotech...

Pharmaceutical Biotechnology Introduction: Principles and Applications Lecture 1 Assistant Prof. Dr. Mohammed Talib Ph.D. in Biotechnology College of Pharmacy the University of Misan What is biotechnology ? Biotechnology : As a term was coined between 1917-1919 by the Hungarian scientist Karoly Erkey. Definitions : It’s the use of biological processes, organisms, or systems to manufacture products intended to improve the quality of Human, animal, and plant life. OR It means the manipulation (as through genetic engineering) of living organisms or their components to produce useful products such as novel pharmaceuticals for example monoclonal antibodies and Covid-19 Karoly Erkey ‘The father of biotechnology’ vaccinations. 2 3  Pharmaceuticals and Biotechnology- Benefits of Combination 1. Targeted and Personalized Medicine Biotech enables the development of drugs tailored to individual genetic profiles, improving treatment precision and minimizing side effects. 2. Innovative Treatment Options Introduces advanced therapies like monoclonal antibodies, gene therapies, and RNA-based treatments for diseases that were previously difficult or impossible to treat. 3. Faster Drug Development Biotechnological tools like genomics and proteomics accelerate drug discovery and development, reducing time to market. 4. Effective Vaccines Technologies like mRNA vaccines (e.g., for COVID-19) demonstrate the ability to respond to emerging infectious diseases rapidly. 5. Addressing Rare and Chronic Diseases The combination enables solutions for rare diseases and chronic conditions through biologics and advanced pharmaceuticals. 6. Cost Efficiency and Scalability Biotechnology-driven production methods reduce manufacturing costs for complex drugs and make large-scale production feasible. 7. Improved Drug Efficacy and Safety Biopharmaceuticals, designed with precision, enhance drug effectiveness while reducing risks of adverse effects. 4  Pharmaceuticals and Biotechnology- Benefits of Combination -When the two disciplines- pharmaceuticals and biotechnology- come together, they result in many advantages for humankind in terms of healthcare. This is possible through Pharmacogenomics (derived from 'pharmacology' and 'genomics') which refers to the study of how the genetic inheritance affects individual human body's response to drugs. 8.Biopharmaceutical drugs aim at designing and producing drugs that are adapted to each person’s genetic makeup. Thus pharmaceutical biotechnology companies may develop tailor-made medicines for maximum therapeutic effects. 9.Also, biotechnology drugs can be given to the patients in appropriate dosages as the doctor would know the patient’s genetics and how the body processes and metabolizes a medicine. 5  Pharmacogenomics Pharmacogenomics is the study of the role of the genome in drug response. Reflects the combination of pharmacology and genomics It analyses how the genetic makeup of an individual affects his/her response to drugs It deals with the influence of acquired and inherited genetic variation on drug response in patients by correlating gene expression or single nucleotide polymorphisms (SNPs) (Drug absorption, distribution, metabolism, and elimination) Pharmacogenetics focuses on single drug-gene interaction, while pharmacogenomics encompasses a more genome wide association approach, incorporating genomics and epigenetics via study the of several genes on drug response. 6  Red Biotechnology Pharmaceutical Biotechnology Biotechnology is a relatively novel and advancing field in which the principles of biotechnology are applied to the development of drugs. Thus, biotechnology products broadly refer to biopharmaceutical drugs generated through research in cell biology, genetics, and recombinant DNA technology The aim of the pharmaceutical biotechnology is to design, produce drugs that are adapted to each person’s genetic makeup, which can give the maximum therapeutic effect 7 Gene Expression – Central Dogma of the life 8  Gene expression Is the process by which the instructions in our DNA are converted into a functional product, such as a protein (the gene has been turned on / or expressed. The products are often proteins, but in non- protein coding genes such as rRNA or tRNA genes…in this case the product is a functional RNA. 9 Transcription: The process of making an RNA copy of a gene's DNA sequence. This copy, called messenger RNA (mRNA), carries the gene's protein information encoded in DNA. Translation: Is the process where ribosomes synthesize proteins using the mature mRNA transcript produced during transcription. 10 Pharmaceutical Biotechnology Products Antibodies: are proteins produced by white blood cells and are used by the immune system to identify bacteria, viruses, and other foreign substances and to fight them off Monoclonal antibodies Is one of the most exciting developments in biotechnology pharmaceuticals during recent years Typically made by fusing myeloma cells with the spleen from a mouse immunized with the desired antigen. e.g orthoclone okt3 (muomomab) reversal acute kidney transplant rejection. How monoclonal antibodies work 11 12 Recombinant DNA products Examples : - Recombinant DNA Vaccines Recombinant DNA Drugs Recombinant DNA Enzymes Recombinant DNA Growth Hormones Recombinant DNA Insulin Recombinant DNA Proteins Recombinant DNA Yeast 13 Recombinant DNA 14 15 Vaccines mRNA vaccines (examples) Moderna Pfizer DNA vaccines (examples) Sinopharm AstraZeneca ZyCoV-D NB: Biotechnology companies in the USA, Europe, and China who were made COVID-19 vaccines. 16 Recombinant DNA Vaccine Plasmid DNA encoding the desired antigen is administered parenterally. The foreign protein is expressed by the host cell and generate an immune response. 17  What are mRNA vaccines and how do they work? mRNA vaccines are a type of vaccines that uses messenger RNA (mRNA) to instruct cells in the body to produce a protein that triggers an immune response. This protein is typically a part of a virus, such as the spike protein of the SARS-CoV-2 virus that causes COVID-19 The Mechanism of Action of mRNA vaccines 1) This type of vaccine gives your cells instructions for how to make the S protein found on the surface of the COVID-19 virus. 2) After vaccination, your muscle cells begin making the S protein pieces and displaying them on cell surfaces. 3) This causes your body to create antibodies. 4) If you later become infected with the COVID-19 virus, these antibodies will fight the virus. 18 19 Recombinant Protein Vaccine Incorporation of the corresponding peptide sequence into a plasmid and expressed in host cells 20 DNA (Gene) Therapy Definition: Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:- Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or “knocking out,” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. 21 How does gene therapy work? A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of viruses, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome. 22 23 24 The Role of PCR and qPCR in Pharmaceutical Sciences 25 qPCR Technique 26  The key benefits of PCR and qPCR in pharmaceutical sciences include: 1. Accurate Genetic Analysis: Detect genetic mutations, polymorphisms, and biomarkers for drug target identification and personalized medicine. 2. Rapid Disease and Pathogen Detection: Identify infectious agents and monitor disease progression to guide drug development and treatment. 3. Drug Efficacy and Mechanism Studies: Analyze gene expression and biological pathways to assess drug effectiveness and mechanisms of action. 4. Therapeutic Monitoring: Quantify viral loads (e.g., HIV) and detect resistance genes for optimizing treatment strategies. 5. Biopharmaceutical Quality Control: Detect contamination and ensure batch consistency in biologics production. 6. Clinical Trials and Drug Development: Validate biomarkers and support companion diagnostics for stratified patient groups. 7. High Sensitivity and Quantification: 27 Detect low levels of DNA/RNA and provide precise quantitative data for research and diagnostics. The course /or syllabus reference 28  Course Assessment 25 Grade for the theoretical exam (Mid-Semester) 5 Grade for active participation in lectures discussions and attendance. 70 Grade for the final exam 29 30