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Drug Discovery Glossary
back to Drug Discovery Tutorial

A  B  C  D  E  F  G  H  I  J  K  L  M  N  O  P  Q  R  S  T  U  V  W  X  Y  Z

ADMEback to top
Absorption, distribution, metabolism, and excretion.

Analogback to top
A compound that is analogous to another. Usually analogs share a common structural scaffold but bear different chemical substituents.

Antigenback to top
A foreign molecule (antigen) triggering the production of an antibody (also called immunoglobulin) by the immune system of an organism.

APIback to top
Active Pharmaceutical Ingredients

Apoptosisback to top
A program of biological functions leading to cell death, also called programmed cell death.

Arrayingback to top
A numbered series of test objects such as activity assays, DNA probes, antibodies, chemical reactions, etc., that are of the same size and/or type.

Arraysback to top
A numbered series of test objects such as activity assays, DNA probes, antibodies, chemical reactions, etc., that are of the same size and/or type.

Assay plateback to top
A plate with typically 96, 384, or 1536 similar microliter volume wells arranged in a two-dimensional array for parallel or multiple tests or reactions.

Attritionback to top
Reduction of lead or drug candidate molecules due to failures in preclinical or clinical trials.

AUCback to top
Area under curve is a measure for drug exposure. AUC is the product of the drug concentration in the bloodstream or a specific tissue and a defined time period.

Binding pocketback to top
A depression, tunnel or cleft where ligands bind on a protein/receptor surface.

BUNback to top
Blood urea nitrogen. A measure of the urea level in the blood. Urea is cleared by the kidney. BUN is a laboratory blood test to assess how well the kidney is functioning. Diseases or toxicity of compounds can compromise kidney function which frequently leads to increased levels of BUN.

CADDback to top
Computer-assisted drug design. Use of computational tools and algorithms for the visualization, design and optimization of leads and drug molecules.

Catalysisback to top
The chemical or biological process whereby the presence and/or participation of an external catalyst molecule (i.e., enzyme), causes a chemical reaction to occur or to improve reaction performance without ultimately altering the catalyst molecule.

Catalyticback to top
The chemical or biological process whereby the presence and/or participation of an external catalyst molecule (i.e., enzyme), causes a chemical reaction to occur or to improve reaction performance without ultimately altering the catalyst molecule.

CDERback to top
Center for Drug Evaluation and Research. Branch of FDA responsible for policy decisions concerning drugs.

Cell lineback to top
Cells which grow and replicate continuously outside the living organism and are maintained in vitro for medical and/or research purposes.

Cell migrationback to top
The biological process of cell movement triggered by a specific stimulus.

Chemical libraryback to top
A collection of distinct, defined and characterized molecules or mixtures thereof.

Chromatographyback to top
The separation of a mixture of substances by charge, size, or other property by allowing the mixture to partition between a moving phase and a stationary phase.

Classback to top
A gene or protein family/class refers to genes or proteins that have in common one or any number of characteristics such as sequence homology, biological function or mechanism of action, etc.

CMCback to top
Chemistry, manufacturing and control. Term used in the context of production and quality control of drugs or drug candidates.

CNSback to top
Central nervous system.

COGSback to top
Cost of goods. The cost of producing a certain amount of a drug molecule.

Compoundback to top
A defined and discreet molecule. A compound can either be a small molecule, a protein or antibody, or an oligonucleotide.

Compound libraryback to top
A collection of distinct, defined and characterized molecules or mixtures thereof.

Compound seriesback to top
A number of analogs which bear common features and were synthesized to address a specific drug discovery or development question.

Conformationback to top
The three-dimensional or spatial orientation of bonds and atoms in a molecule. A certain molecule usually exists in a number of possible conformations.

Controlled trialback to top
Design of a clinical trial that includes a balanced and randomized group of test subjects in terms of age and gender (where applicable), a control group following the same test protocol without actually receiving drug (placebo), and an independent review of the trial data. Trials may be open, blind or double-blind.

Cytotoxicityback to top
Toxic to cells.

Disease markerback to top
A direct or indirect (surrogate) readout measuring the degree, reversal or progression of a specific disease.

DNAback to top
Deoxyribonucleic acid. The chemical building block of the genetic information in the cell, specifying the characteristics of most living organisms. DNA is usually in the form of two complementary strands.

Drug targetback to top
Biological molecule or molecules that are inhibited, activated or modulated by a drug molecule. Drug targets can be any form or combinations of (glyco)proteins, DNA or RNA.

Dynamic rangeback to top
The specifications under which an assay measurably responds to the interference by a test compound.

EC50back to top
The EC50 represents the plasma concentration/AUC required for obtaining 50 percent of the maximum effect in vivo.

ED50back to top
The dose of a drug that is pharmacologically effective for 50 percent of the population exposed to the drug or a 50 percent response in a biological system that is exposed to the drug.

Effectivenessback to top
Power or capacity to produce a desired, or in the context of disease, beneficiary effect.

Efficacyback to top
Power or capacity to produce a desired, or in the context of disease, beneficiary effect.

Enzymeback to top
A protein catalyzing a chemical reaction. Biological processes are to a high degree driven by enzymatic reactions.

Exposureback to top
Exposure measures the interaction/contact of an organism and a compound over a specified time period. The area under the curve (AUC) is an important parameter for exposure. It is a measure of how much of a drug reaches the bloodstream in a set period of time. AUC is calculated by plotting drug blood concentration at various times during a 24-hour or longer period and then measuring the AUC between 0 and 24 hours.

False negativeback to top
False positive: a test result that wrongly shows an effect to be present. False negative: a test result within specifications that wrongly shows no effect to be present.

False positiveback to top
False positive: a test result that wrongly shows an effect to be present. False negative: a test result within specifications that wrongly shows no effect to be present.

Familyback to top
A gene or protein family/class refers to genes or proteins that have in common one or any number of characteristics such as sequence homology, biological function or mechanism of action, etc.

FDAback to top
Food and Drug Administration. US regulatory agency (http://www.fda.gov) responsible for the evaluation and approval of drugs and medical devices.

Formulationback to top
The list of chemicals/substances that are not the therapeutically active ingredient, and their relative amounts, to be used in the preparation of a drug.

Functionback to top
Biological/physiological role of a gene, gene product (protein, enzyme). Functional assays assess the this role.

Functionalback to top
Biological/physiological role of a gene, gene product (protein, enzyme). Functional assays assess the this role.

Gain-of-functionback to top
Restoration of the function of a gene or gene product. Usually through complementation with a gene or protein, sometimes through the action of a compound.

Gene expressionback to top
Degree to which a certain gene is activated and transcribed into gene products. Measured by the amount of RNA or protein.

Gene productback to top
Protein or enzyme encoded in a gene. Some genes can code for more than one gene product due to gene-splicing post-translational modifications.

Gene silencingback to top
Suppression of a gene and its function.

Genomicsback to top
The study of the sequence, structure, and function of the genome.

Genotoxicityback to top
A measure of the potency of adverse effects of a toxin on DNA.

GLPback to top
Good laboratory practices.

GMPback to top
Good manufacturing practices.

GPCRback to top
G-protein coupled receptor.

GRASback to top
Generally recognized as safe.

hERG-channelback to top
Human ERG-channel. hERG is an acronym for "human ether-a-go-go related gene" and names a pore-forming subunit of a family of potassium ion channels in heart cells.

Heteroatomback to top
Atoms differently than carbon (C) Examples are nitrogen (N), oxygen (O) or sulphur (S).

Heterooligomerback to top
A macromolecular complex composed of two or more subunits which are different from each other.

Hitback to top
A compound that shows activity in a primary screen.

Homologback to top
This is a gene, gene product or compound that shares a common ancestry or morphology with a different gene, gene product or compound.

Homologyback to top
This is a gene, gene product or compound that shares a common ancestry or morphology with a different gene, gene product or compound.

IC50back to top
The IC50 represents the concentration of a drug that is required for 50 percent inhibition in an assay.

Imagingback to top
Procedures that produce pictures of areas inside a cell, a tissue or the body of an organism.

In silicoback to top
Computationally as opposed to in vitro or in vivo.

INDback to top
Investigational new drug.

Inhibitionback to top
A chemical compound that has the effect of blocking or slowing a biological process, for example an enzymatic reaction.

Inhibitorback to top
A chemical compound that has the effect of blocking or slowing a biological process, for example an enzymatic reaction.

Kinaseback to top
Enzyme family catalizing phosphorylation, i.e., addition of a phosphate group to its substrate.

Knock-inback to top
A type of targeted mutation in which an alteration in gene function other than a loss-of-function is produced.

Knockoutback to top
A type of targeted mutation in which loss-of-function is produced.

LD50back to top
LD50: (Lethal Dose 50) is the dose of a chemical which kills 50 percent of a sample population. In full reporting, the dose, treatment and observation period should be given. LD50s are strictly only comparable when the age, sex, and nutritional state of the animals is specified.

Leadback to top
A compound with a confirmed activity profile that warrants development.

Lead seriesback to top
A number of analogous compounds synthesized around a confirmed active—the lead.

Ligandback to top
A compound, mostly a small molecule, that binds to a receptor/protein.

Liquid handlingback to top
Transfer and mixing of solutions containing reagents for an assay or reaction, usually in the nanoliter to milliliter range.

Lysateback to top
Solution which results from cell disintegration (lysis) due to membrane rupture or the breakdown of the cell wall.

Lyse/Lysisback to top
Cell disintegration due to membrane rupture or the breakdown of the cell wall.

Lysisback to top
Solution which results from cell disintegration (lysis) due to membrane rupture or the breakdown of the cell wall.

Metabolismback to top
The biochemical processes that sustain a living cell or organism. Usually divided into two types: catabolism, the breakdown of complex substances into simple ones, with the release energy; and anabolism, the building up of complex substances from simpler ones, with the absorption or storage of energy. Molecules that are part of these processes are metabolites.

Metaboliteback to top
The biochemical processes that sustain a living cell or organism. Usually divided into two types: catabolism, the breakdown of complex substances into simple ones, with the release energy; and anabolism, the building up of complex substances from simpler ones, with the absorption or storage of energy. Molecules that are part of these processes are metabolites.

Mitosisback to top
The process of cell division. Post-mitotic cells have stopped to divide.

MoAback to top
Mechanism of action.

Molecular targetback to top
Molecule with a function in disease that is targeted with a compound to achieve a therapeutic effect.

Morphologyback to top
The form and structure of an organism.

MTDback to top
Maximum tolerated dose.

MTT-likeback to top
The MTT assay is a spectroscopic laboratory test for measuring cell growth based on 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium salts (MTT).

Mutationback to top
A change in a gene that can occur randomly (naturally) or that can be deliberately caused in the laboratory by scientists.

NCEback to top
New chemical entity.

NDAback to top
New drug application.

Oligonucleotideback to top
A short sequence (usually 2-50 bases) of DNA or RNA.

Pathwaysback to top
A system of proteins that work together. For example, a pathway could include protein A which sends a signal to protein B, which sends a signal to protein C, and so on until a biological effect occurs. Biological pathways are often complex, have many branching points and regulatory feedback loops.

PD-Pharmacodynamicsback to top
The study of drug action on living organisms.

Pharmacologyback to top
The study of drugs and their origin, nature, properties and effects upon living organisms.

Pharmacophoreback to top
The three-dimensional arrangement of atoms, or groups of atoms, responsible for the biological activity of a drug molecule.

Phenotypeback to top
A phenotype is the visible properties of an organism that are produced by the interaction of the genes and the environment.

Phosphorylationback to top
The addition of a phosphate group to proteins or DNA/RNA.

PK-Pharmacokineticsback to top
The study of the action of an organism on a drug. Usually encompasses absorption, metabolism, distribution and excretion (ADME).

Post-mitoticback to top
The process of cell division. Post-mitotic cells have stopped to divide.

Pro-drugback to top
Originates from the term "progenitor drug." A compound that is converted within the body into the active form that has therapeutic effects.

Proteinback to top
A large biomolecule composed of one or more chains of amino acids in a specific order. Proteins are required for the structure, function, and regulation of cells, tissues, and organs.

Protein-protein interactionsback to top
Describes the mostly complex nature of specific and selective binding of proteins.

QSARback to top
(Quantitative) structure-activity relationship. A model describing how a pharmacophore effects its target.

Radioisotopeback to top
A radioactive form of an element. In research radioisotopes are widely used to label specific molecules, which can be detected by the radiation they emit.

Receptorback to top
A specific protein-binding site on a cell's surface or interior. When compounds bind to receptors, various cellular functions are activated or inhibited.

Recombinantback to top
Produced by genetic engineering or altered in the genes. In a recombinant organism one or more genes which can be endogenous or exogenous have been mutated, added, or deleted.

Resistanceback to top
The degree of unresponsiveness of a disease-causing microorganism or cell to drugs (for example, penicillin-resistant bacteria).

Ribosomeback to top
A subcellular particle composed of RNA and protein, which is part of the protein-synthesizing machinery of the cell.

RNAback to top
Ribonucleic acid, molecules involved in transcribing genetic information for the synthesis of proteins stored in DNA and then translating this information into the sequence of amino acids in the protein.

SARback to top
(Quantitative) structure-activity relationship. A model describing how a pharmacophore effects its target.

SBDDback to top
Structure-based drug design.

Scale-upback to top
Describes the process of achieving production in a larger scale, i.e., from the gram to kilogram scale or from microliter to liter scale.

Screenback to top
In drug discovery testing large numbers of compounds in order to identify those with particular characteristics.

Screeningback to top
In drug discovery testing large numbers of compounds in order to identify those with particular characteristics.

Sequencingback to top
Determination of the order of nucleotides in a DNA or RNA molecule, or the order of amino acids in a protein.

Serial dilutionback to top
Systematic dilution of a reagent by a fixed dilution factor resulting in a series of concentrations.

Side effectback to top
An undesirable effect of a drug.

Signal transductionback to top
The way by which a cell relays information from receptor binding to cellular response.

Small molecule drugsback to top
Therapeutic molecules with a molecular weight below 1 kDa. A typical small molecule drug has a molecular weight between 250 and 400 Da.

Solvationback to top
The process by which solvent molecules surround and interact with solute ions or molecules.

Spectroscopyback to top
The study of the interaction between electromagnetic radiation (light) and matter.

Substrateback to top
The molecule upon which an enzyme acts.

Susceptibilityback to top
The degree to which an organism is sensitive to a therapeutic or a disease.

Target classback to top
Systematic or empirical name of a target family, i.e., kinases.

Throughputback to top
Measure for the number of assays or data points per time unit, i.e., 1,000 assays per day.

Transgenicback to top
A genetically altered organism that stably incorporates genes from another organism and can pass them on to succeeding generations.

Upregulationback to top
An increase of expression, transcription, and/or translation of a cellular protein or gene.

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