Summary Biopharmaceuticals and Biotechnology: A Simplified Guide
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Course
Pharmacology
Institution
Pharmacology
This simplified guide gives an overview of key concepts in biopharmaceuticals and biotechnology, covering how these complex drugs are made, the challenges involved, and the role of personalized medicine in improving patient outcomes.
Biopharmaceuticals and Biotechnology: A Simplified Guide
1. Introduction to Biopharmaceuticals
Biopharmaceuticals, or biologics, are drugs made using biological processes, unlike traditional
medicines made through chemical synthesis. These drugs are often more complex and larger in
size, involving proteins, cells, or genetic material. They’re produced using living organisms, like
bacteria or mammalian cells, and include treatments such as monoclonal antibodies, vaccines,
and gene therapies.
1.1 Monoclonal Antibodies (mAbs)
Monoclonal antibodies are lab-engineered proteins designed to target and bind to specific cells,
such as cancer cells or pathogens. They are made from identical immune cells cloned from a
single parent cell.
Examples:
Rituximab: Used to treat certain cancers and autoimmune diseases by targeting B cells.
Trastuzumab (Herceptin): Targets the HER2 protein in breast cancer cells, helping
slow cancer growth.
How They Work: Monoclonal antibodies fight diseases by:
1. Binding directly to harmful cells (e.g., viruses or cancer cells).
2. Recruiting the immune system to attack the target cells.
3. Blocking specific receptors or pathways that allow diseases like cancer to progress.
Uses: Monoclonal antibodies are commonly used in cancer treatment, autoimmune disorders,
and infectious diseases.
1.2 Vaccines
Vaccines teach the immune system how to fight infections by introducing parts of viruses or
bacteria (called antigens). This triggers an immune response, helping the body recognize and
fight off the actual pathogen if encountered later.
Examples:
mRNA COVID-19 vaccines (Pfizer-BioNTech and Moderna): These use messenger
RNA to instruct cells to make a viral protein, prompting an immune response.
, Hepatitis B vaccine: Contains a viral protein (antigen) to stimulate immunity without
using a live virus.
How They Work: Vaccines expose the immune system to a harmless form of a pathogen,
training it to recognize and combat the disease in the future.
1.3 Gene Therapy
Gene therapy is a method of treating diseases by altering a person’s genes. It involves adding,
removing, or editing genetic material in cells to correct or replace defective genes that cause
diseases.
Examples:
Luxturna: Treats inherited blindness by delivering a working version of a gene into eye
cells.
Zolgensma: A gene therapy for spinal muscular atrophy, replacing the faulty SMN1 gene
to restore muscle function.
How It Works: Gene therapy can work by:
1. Replacing faulty genes with healthy ones.
2. Silencing harmful genes through RNA interference or other techniques.
3. Editing specific parts of the genome using tools like CRISPR to correct genetic
mutations.
2. How Biopharmaceuticals Are Made
Biopharmaceuticals are produced using living cells, making the process more complicated than
making traditional chemical drugs. It involves several steps: manipulating DNA, growing cells,
and purifying the final product.
2.1 Recombinant DNA Technology
This technology combines genes from different organisms to make proteins or other therapeutic
molecules.
How It Works:
1. Gene Cloning: The gene that codes for the desired protein (like insulin) is isolated and
inserted into a vector (a DNA molecule that can replicate in cells).
2. Transformation: This vector is then introduced into host cells (like bacteria or
mammalian cells) that will produce the desired protein.
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