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Pharma & Biotech

How to speak biotech: An A-Z guide

Biotech is a broad church. The term can be applied to any business that manufactures or uses products with a biological base – and it can be difficult to keep up with the many scientific buzzwords in this rapidly expanding field. Help is at

If you thought mining terminology was a minefield, the rich vein of biotech buzzwords will have you reaching for the dictionary.

It can be difficult to keep up with biotech terms when the field - fuelled by a raft of discoveries in genetics, biochemistry, molecular biology and other sciences that fall into the category - is expanding as quickly as the universe itself.

Biotech originated with the study of living organisms, which covers a vast array of sectors including agriculture, medicine, food and industrial technology.

The term can be applied to any business that manufactures or uses products with a biological base – this may include textiles, wood pulp products, food, biofuels and therapeutic drugs.

Gene technology drives a large part of modern biotech, with new technologies and products being developed every year.

This by-no-means complete list is designed to help the average investor distinguish their alleles from their antigens, and navigate the world of biotechnology.

Active immunity: A type of immunity where resistance to a disease is built up by either having the disease or receiving a vaccine for it.

Allele: One of two or more versions of a gene – an individual inherits two alleles for each gene, one from each parent. If the two alleles are the same, the individual is homozygous for that gene.

Antibody: A protein produced by the immune system that binds to a specific antigen.

Antigen/immunogen: A substance, usually a protein, that when recognised as 'foreign’ by the immune system will trigger an immune response, stimulating the production of antibodies. When introduced into a vertebrate organism, an antigen is bound by the antibody or a T cell receptor.

Autologous cells: Cells that are taken from an individual, cultured (or stored), and, possibly, genetically manipulated before being introduced back into the original donor.

Biologics: A biotechnology drug, such as a protein expressed by an engineered cell, or an antibody produced using recombinant DNA.

Biomanufacturing: The use of living cells to produce a biological product – for example a therapeutic protein that might be used in cancer treatment.

Biomarker: A measurable physiological event, such as the expression of a gene. Biomarkers are often used to indicate the presence or progression of a disease.

Biosimilar: A medicine or biologic product achieved using a different process than the one that originally produced the effect, but which has a similar structure and function.

Blind trial: A trial where the participants do not know which treatment or intervention they have been allocated.

Cell: The building blocks of any living organism. Each cell typically contains genetic material, an energy-producing system, and protein-making ability, encased in a membrane.

CAR T cell therapy: Chimeric antigen receptor (CAR) T-cell therapy uses immune or white blood cells, called T cells, to fight cancer by altering them so they can find and destroy cancer cells.

Companion diagnostic: A diagnostic used by physicians to inform their prescribing decision.

CRADA (cooperative research and development agreement): A deal in which two companies agree to join resources to move a product concept or technology ahead – big pharma will often partner with emerging biotech companies in a CRADA.

CRO (clinical research organisation): A supplier firm that offers contract services in testing, clinical trials and manufacturing.

Cytokinesis: Cytoplasmic division and other changes exclusive of the nuclear division that occurs through mitosis or meiosis.

Cytology: The study of the structure and function of cells.

Deoxyribonucleic acid (DNA): DNA is a double-stranded helix held together by bonds between pairs of nucleotides – the molecule that encodes genetic information.

Diagnostic: A test used to identify the presence of a disease or disorder, or to monitor the progression of treatment. A routine diagnostic is a broad screening tool; a specialty diagnostic screens for a specific disease.

Di-sulphide bond: A chemical bond that stabilises the three-dimensional structure of proteins, and therefore the protein's normal function.

Endpoint: In clinical trials, an event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial. Endpoints are usually identified in the study objectives.

Epigenetic modification: Chemical modification made to a nucleotide base. Epigenetic modification can change the degree to which a gene is expressed. This occurs as part of normal development but irregularities are associated with various diseases.

Fission: Asexual reproduction which involves the division of a single-celled organism into two new equally sized single-celled individuals.

Fusion protein: A protein that is expressed by a gene developed through recombinant DNA methods from parts of genes encoding two or more proteins.

Genetically Modified Organism (GMO): Any organism whose DNA has been altered using genetic engineering techniques.

Genome: All of the genetic material in the chromosomes of a particular organism.

Genomic editing: A type of genetic engineering in which DNA is inserted, replaced or removed from a genome.

Haploid: A cell with half the usual number of chromosomes, or only one chromosome set. Sex cells are haploid.

Hapten: A small molecule which, when chemically-coupled to a protein, acts as an immunogen and stimulates the formation of antibodies not only against the two-molecule combination but also against the hapten.

Homozygous: Having two identical alleles for a particular gene.

Inducer: A substance that increases the rate of enzyme synthesis, usually by blocking the action of the corresponding repressor.

Immune checkpoint inhibitors: Therapeutics that lift the inhibitions on T-cells in order to make them more likely to attack cancer cells.

INK (invariant natural killer) T cells: Cells that recognise lipid antigens presented by a cell-surface molecule known as CD1d. They have been shown to have important roles in many diverse immune responses.

Knock out: A technique used to inactivate a particular gene in order to define its function.

Large-molecule drug: Protein-based therapeutics – these contain molecules that are too large to enter cells.

Leukocyte: A colourless cell in the blood, lymph and tissues that is an important component of the body’s immune system, which is also known as a white blood cell.

Lipoproteins: A group of serum proteins that transport fats and cholesterol in the bloodstream. Abnormalities in lipoprotein metabolism can feature in heart diseases.

Master cell bank: A single pool of cells that generally has been prepared from the selected cell clone under defined conditions.

Microbiome: An ecosystem of microbes, their genomes and environmental interactions in a particular environment.

Mitosis: Process of cell reproduction whereby the daughter cells are identical in chromosome number to the parent cells.

Meiosis: Meiosis is the division of a germ cell into four cells, each with half the number of chromosomes of the parent cell.

Monoclonal antibody: An antibody produced by a single clone of cells, which therefore consistently binds to the same epitope of an antigen.

Mutation breeding: Genetic change caused naturally or using mutagens. Stable mutations in genes are passed on to offspring, while unstable mutations are not.

Nanotechnology: The understanding and control of matter at dimensions between approximately 1 and 100 nanometres, with myriad applications across science, energy and even defence.

NDA: A new drug application is the mechanism by which drug companies formally propose that the US Food and Drug Administration (FDA) approve a new pharmaceutical for sale and marketing.

Nucleotide: A nucleotide is the basic building block of nucleic acids. RNA and DNA are polymers made of long chains of nucleotides.

Nutraceutical: Term coined by the Foundation for Innovation in Medicine in 1991 referring to “any substance that may be considered a food or part of a food and provides medical or health benefits, including the prevention and treatment of disease”.

Orphan drug: Drug developed for a condition that affects fewer than 200,000 individuals (in the US).

Peptide: Peptides are short strings of amino acids, typically comprising up to 50 amino acids. Amino acids are also the building blocks of proteins but peptides may be easier for the body to absorb than proteins because they are smaller and more easily broken down.

Pharmacodynamics: The study of the effect of a drug on the body as it relates to increasing dose.

Pharmacogenomics: The study of the correlation between an individual’s genetic make-up and their response to drug treatment. Some drugs work well in some patient populations and not others.

Pharmacokinetics: The study of the time course of drug’s lifespan in the body, from absorption to distribution, metabolism and excretion.

Phase 1 clinical trials: The first stage of the testing of a new drug or process on human participants. Investigators are interested in learning about safety, side effects and dosage levels.

Phase 2 clinical trials: Small trials on people who have the medical condition under investigation. The objective is to determine whether the experimental drug provides a beneficial effect. There is usually a control group the receives a placebo alongside the treatment group who receive the drug.

Phase 3 clinical trials: Larger, carefully controlled long-term studies on patients to determine whether the drug will be effective in normal medical settings. During Phase 3, information is gleaned on long-term side effects and safety.

Pre-clinical testing: This generally means testing in animals, which in medicine generally happens as a precursor to Phase 1 to 3 testing.

Quantitative Reverse Transcriptase PCR: A technique used to measure the levels of gene expression of a particular gene.

Rational drug design: The development of therapeutic drugs based on an understanding of the underlying disease mechanism.

Receptor: A region of tissue found on the surface of a cell that responds to a specific chemical messenger, such as a neurotransmitter, antigen or hormone.

Recombinant: Relates to an organism, cell, or genetic material formed by recombination.

RNA: Ribonucleic acid (RNA) is a molecule similar to DNA. Molecular biology suggests that the primary role of RNA is to convert the information stored in DNA into proteins.

RNA interference (RNAi): A technique used in science to block the expression of a particular protein.

Selective medium nutrient: Matter that will support the growth of specific organisms while inhibiting the growth of others.

Sequence: The order of nucleotides in a DNA or RNA molecule, or the order of amino acids in a protein.

Signalling molecule: A molecule that binds to a cellular receptor, which results in the cell starting or stopping the production of a particular protein.

Small molecules: Molecules made by chemistry into traditional pills, or by training cells to produce proteins that can be used as medications.

Spike protein: A spike protein, used in vaccines, is a protein that forms a large structure known as a spike or peplomer projecting from the surface of the enveloped virus. It is located on the outside of the virus so the immune system can recognise it easily.

Stem cell: Stem cells are undifferentiated human cells that have the ability to self-renew and develop into many different cell types in response to extracellular cues.

T-Cell: An immune system cell that can be used to recognise specific pathogens because of the shape of its cell-surface receptor.

Target validation: Determining if targeting a particular molecule that might be involved in a disease mechanism will be a safe and effective means of therapy.

Transgenic organism: An organism whose genome has been altered by the incorporation of foreign DNA.

Unipotent: A molecule capable of developing into only one type of cell or tissue.

Unblinded results: Unblinding, sometimes referred to as code-break, is the process by which the treatment details that form part of a study are made available either on purpose or by accident.

Upstream processing: The phase of biomanufacturing consisting of establishing cell banks and seeding and scaling up cell cultures.

Viral vaccines: Vaccines consisting of live rather than dead or separated parts of viruses. The virus itself is genetically engineered so that it elicits the immune response to the viral pathogen without causing the disease itself.

Vector: A vehicle for the transfer of DNA from one organism to another.

Working cell bank: A vialed collection of cells that are derived from the master cell bank.

X-chromosome: A chromosome associated with sex determination - the female has two X-chromosomes, while the male has one X-chromosome and one Y-chromosome.

Xenobiotic: A chemical compound not produced by living organisms – a manufactured chemical compound.

Y-chromosome: The partner of the X-chromosome in the male of many animal species.

Zygote: A diploid cell formed by the fusion of two haploid gametes during fertilization – the first cell of the new organism.

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