GLOSSARY

Skip To: A B C D

A

antibody
A protein substance produced by the immune system and found in the blood or tissues in response to a specific antigen, such as a bacterium or toxin.
antigen
Any material that causes the immune system to react when it comes into contact with human or animal tissues.
apoptosis
Programmed cell death, a natural process that the body uses to dispose of old, damaged or unwanted cells.
assay
A test to determine whether compounds (drugs, chemicals, etc.) have the desired effect either in a living organism, outside an organism, or in an artificial environment.

B

biologics license application (BLA)
Biologics License Application, a document filed with the US Food and Drug Administration (FDA) as part of the late-stage approval process for biotherapeutic products, such as proteins and antibodies.

C

combinatorial chemistry
Automated technology that allows chemists to synthesize (manufacture) small quantities of different compounds that share a similar chemical structure.
computational chemistry
Computer-assisted techniques that enable chemists to understand and design compound structures in the context of a biological target.
cytoplasm
The jelly-like substance that surrounds the nucleus of a cell.

D

database mining
A highly automated process that compares the recorded sequence for each identified gene to all known genes; results are stored in an annotated database.
diagnomics
Molecular diagnostic testing that gives clinicians information about patients that can be used in making medical decisions.
deoxyribonucleic acid (DNA)
The molecule present in every cell of the body that contains genetic information; a double-stranded molecule, shaped like a twisted ladder, consisting of four nucleotides in varying sequences that are paired with one another. The nucleotides, represented by the letters A, C, G, and T, are the building blocks of DNA. DNA is composed of a series of genes linked by non-gene regions.
drug development process
  • Discovery: Identification of a biological, genetic or protein target linked to a particular disease, and subsequent lead identification of a potential drug that interacts with the target to help cure the disease or halt its progression.
  • Pre-clinical: Comprehensive in vitro (lab dish) and animal testing of the drug candidate to establish its target specificity, toxicity in various doses and pharmacokinetics.
  • Clinical Phase I: Human trials conducted with small numbers of patients to demonstrate the safety and effectiveness (efficacy) of an experimental drug or procedure. Tests are conducted to establish dosage, side effects and pharmacokinetics.
  • Clinical Phase II: Trials with small numbers of patients conducted to identify drug performance characteristics (optimal dosing, administration, key indication).
  • Clinical Phase III: Pivotal trials conducted with larger patient populations to establish efficacy and provide additional safety information.
  • Approval: Data are analyzed and submitted for regulatory review. The US submission to the FDA is called an NDA (New Drug Application) or BLA (Biologic License Application); in Europe, data are submitted to the EMEA (European Medicines Evaluation Agency) in a submission called an MAA (Marketing Authorization Application). After stringent analysis and review of the submission, the regulatory agency provides final approval.
Skip To: E F G H I J K L

E

expression profiling
A method for comparing genes that are expressed (present and active) in healthy tissue with those expressed in diseased tissue. It can help to identify proteins associated with the disease, narrowing the search for appropriate drug targets.

F

G

gene
The fundamental physical and functional unit of heredity; a DNA segment that codes for (directs) the production of one protein.
genome
The complete set of genes of an organism, including animals, plants, bacteria, viruses, etc. Genome size is generally given as the total number of base pairs in an organism.
genomics
The study of genetic material in the chromosomes of a particular organism and its activity.
genotype
The genetic make-up of an individual organism.

H

high throughput screening
Rapid evaluation of large numbers of chemical compounds to determine which one(s) interact with a given biologic target.

I

informatics
The practice of building and deploying tools that allow researchers to extract information of value from masses of data to design better experiments.

J

K

knockout models
Models, such as laboratory mice, which have had a normal gene replaced by a marker, providing clues as to the missing or "knocked-out" gene's relation to organ development, viability and reproduction.

L

Skip To: M N O P

M

metastasize
In cancer, to spread, by transferring a malignancy (out-of-control growth) from the site of disease to another part of the body.
monoclonal antibody, humanized monoclonal antibody
A highly specific protein that can bind to any single substance to register a presence or to deactivate it. Monoclonal antibodies can be used to detect disease or tag disease cells for attack with drugs, radiation, or toxins.

N

O

oncogene
A gene that makes a normal cell grow abnormally and causes cancer.

P

pharmacokinetics
The behavior of a drug in the body over time.
predictive medicine
The use of diagnomics and pharmacogenomics to help prescribe the most appropriate course of treatment for a patient.
proteasome
A distinct enzyme complex within cells responsible for breaking down proteins that have been marked for disposal by the attachment of a tag called ubiquitin, including regulatory proteins governing processes such as cell division.
proteins
Fundamental components of all living cells, including substances such as enzymes, hormones, and antibodies, that are necessary for the proper functioning of an organism.
proteomics
The study of proteins, how they are produced, how they interact, and what they do.
Skip To: Q R S

Q

R

ribonucleic acid (RNA)
A nucleic acid that transmits genetic information from DNA to the cytoplasm of a cell. Like DNA (deoxyribonucleic acid), it consists of long chains of four nucleotides, A, C, G, and U (rather than the "T" found in DNA).

S

single nucleotide polymorphism (SNP)
A single erroneous "letter" substitution in a string of DNA. SNPs can serve as unique genetic markers, but they may also form the basis for an individual's susceptibility to a disease or lack of response to a given drug.
small molecule drug
One or more active chemical compounds, typically formulated as an orally available pill, that interact with a specific biological target, such as a receptor, enzyme or ion channel, to provide a curative effect.
specificity
In pharmacology, the ability of a drug to target specific tissues without affecting other tissues. In diagnosis, specificity is the degree to which a test can identify a disease to the exclusion of all others.
Skip To: T U V W X Y Z

T

target validation
Evaluation of a gene's specific function in the disease process.
therapeutic protein
A complex organic compound, composed of one or more chains of amino acids, that has been found to have a curative effect.
thrombosis
The initial stages of the formation of a blood clot, in which specialized blood cells called platelets clump together at the site of a wound or other damage to a blood vessel.
transgenic models
Organisms, such as fruit flies or mice, which carry an extra gene — one that has been over-expressed, providing clues as to the gene's relation to organ development, viability, and reproduction.

U

V

W

X

Y

Z