Glossary

Question 1

A therapeutic target is in general a macromolecule (typically a protein), which may cause or be associated with a particular disease, that can be modulated by a therapeutic agent in a measurable way.

Answer 1

TARGETS OR DRUG TARGETS

Question 2

Are macromolecule, made up of deoxynucleic acid (DNA) based. In humans they vary in size from a few hundred DNA bases to more than 2 million bases and about 20,000 to 25,000 have been identified.

Answer 2

GENES

Question 3

How many genes have been identified?

Answer 3

20,000 to 25,000

Question 4

process of separation and characterization of all the proteins of a biological system. Target identification can be performed by comparing the protein expression levels in normal and diseased
tissues.

Answer 4

PROTEOMICS

Question 5

Diverse molecules from biological origins that include, nucleic-acids, various (recombinant) proteins, antibodies. some types of peptides.

Answer 5

BIOLOGICS

Question 6

typically have a high molecular weight

Answer 6

BIOLOGICA

Question 7

large biomolecules (up to thousands of atoms) that comprise one or more chains of amino acids.

Answer 7

PROTEINS

Question 8

are products of genes; they generally fold into a specific three-dimensional (3D) structure that determines their activities.

Answer 8

PROTEINS

Question 9

short chains of amino acids.

Answer 9

PEPTIDES

Question 10

Some peptides belong to the category of_____ or _____, among others.

Answer 10

GLYCOPEPTIDE
LIPOPEPTIDE

Question 11

Very short and
modified peptides can behave like small molecules while longer peptides (e.g., insulin which is used to manage
diabetes) fit in the category of biologics. At present, there are very few approved peptides that can be given by oral
route but important work is ongoing in this field to enable oral delivery

Answer 11

PEPTIDES

Question 12

Any organic compound with around 80–100 atoms. Most are made synthetically (aspirin), while others can be derived from natural product (e.g., morphine, which is used to relieve moderate to severe pain)

Answer 12

SMALL MOLECULES

Question 13

Small Molecules are organic compound with around______ . Most are made synthetically (aspirin), while others can be
derived from natural product (e.g., morphine, which is used to relieve moderate to severe pain)

Answer 13

80-100 ATOMS

Question 14

Natural product which is used to relieve moderate to severe pain

Answer 14

MORPHINE

Question 15

A molecule suitable for clinical testing. The molecule is expected to bind selectively to a target involved in the disease
process, to elicit the desired functional responses in vivo, often in animal models of the human disease, to have
adequate bioavailability and bio-distribution within the body to reach the intended target and to pass formal toxicity
evaluation in various in vitro and animal models

Answer 15

DRUG CANDIDATES

Question 16

branch of molecular biology that involves extensive analysis of biological data using computers

Answer 16

BIOINFORMATICS

Question 17

field that attempts to solve chemical problems on the computer, including chemical structure coding, properties modeling and development of databases

Answer 17

CHEMOINFORMATICS

Question 18

Combines computer sciences and mathematics and uses (large) datasets to enable problem-solving. It includes various learning approaches, natural language processing, knowledge representation and reasoning, among others

Answer 18

ARTIFICIAL INTELLIGENCE (AI)

Question 19

A drug has to reach the intended target(s), elicit the desired functional response with no or limited toxicity and be eliminated from the body (typically via the liver or
kidneys). These are critical properties of the drug candidates that are commonly investigated at various stages of the
process

Answer 19

ADMET ( ADSORPTION, DISTRIBUTION, METABOLISM, EXCRETION, TOXICITY)

Question 20

Physiologically-based pharmacokinetic modeling and simulation is a computer modeling approach that incorporates blood flow and tissue composition of organs to define the pharmacokinetics of drug candidates

Answer 20

PB/PK

Question 21

the time-concentration profile of drugs administered in vivo to living organisms. parameters include clearance, volume of distribution, peak plasma concentration. . . is sometimes described “what the body does to a drug”

Answer 21

PHARMACOKINETICS

Question 22

efers to the relationship between drug concentration at the site of action and the resulting effect, including the time course and intensity of therapeutic and adverse effects.

Answer 22

PHARMACODYNAMICS

Question 23

parameters include minimum effective concentration, maximum safe concentration, onset of action, therapeutic range and therapeutic index. describes how biological processes in the body respond to or are impacted by a drug

Answer 23

PHARMACODYNAMICS

Question 24

Relationship of the drug effect (pharmacodynamics) to the drug concentrations in the body compartments (e.g., blood, organs) as a function of time after drug administration

Answer 24

PK/PD

Question 25

Action of a drug on targets other than the intended biological target. Such events commonly contribute to adverse effects or toxicity, however, in some cases, off-target activity can be valuable for therapeutic purposes

Answer 25

OFF TARGET ACTIVITY

Question 26

A drug is usually designed to interact with its intended target. In some situations, the drug induces exaggerated and adverse pharmacological effects at the target of interest. This is commonly referred in the literature to as on target toxicity

Answer 26

ON TARGET TOXICITY

Question 27

Unintended pharmacological effects that occur when a medication is administered correctly. There are different types of reactions (mild, moderate or severe) that can be dose-dependent or not

Answer 27

ADVERSE EVENTS

Question 28

Secondary unwanted effects that occurs due to the drug therapy. Side effects are usually known and patients are
informed about such effects

Answer 28

SIDE EFFECTS

Question 29

• Preclinical studies are a stage of research that precedes clinical trials (testing in humans). The therapeutic agents are
  tested in animal models of human diseases or in systems that simulate human diseases.
• The main goals are to
  determine a starting, safe dose for first-in-human study and assess potential toxicity. Research into early formulations (e.g., tablet, capsule, intramuscular injection, intravenous, sublingual. . .) is also performed

Answer 29

PRECLINICAL DEVELOPMENT

Question 30

Research studies performed in humans aiming at evaluating the efficacy (does the drug cure or slow the progression
of a disease?) and safety (does the drug cause undesired effects, or toxicity?) of drug candidates.

Answer 30

CLINICAL TRIALS

Question 31

Aka“first-in-humans” trial. Test on 20–80 healthy volunteers to assess the safety and pharmacokinetics, absorption,
metabolism, and elimination, actions on the body, as well as possible side effects, formulation, and dose. In some
cases, a placebo can be used. For some drugs, a phase 0 can be sometime performed before phase I to evaluate some properties of the drug on few patients or on healthy individual

Answer 31

PHASE I

Question 32

Assesses drug safety and efficacy on about 100–500 patients (suffering from a specific disease), some of which may
receive a placebo or an approved drug for that disease, called“standard of care”. Analysis of optimal dose is performed while adverse events and risks are recorded

Answer 32

PHASE II

Question 33

enrolls numerous patients (e.g., 1,000–5,000), enabling medication labeling and instructions for proper drug use. Efficacy, dose, and toxicity are observed and adjustments to the final medication label are being made based on such information

Answer 33

PHASE III

Question 34

Following drug approval and manufacturing, regulatory agencies require companies to monitor the safety of the approved drug. Drug makers, health professionals, hospitals and patients report adverse events occurring when taking the approved drug

Answer 34

Phase IV or pharmacovigilance or“real world evidence”

Question 35

The dosage (a range of concentrations) of a drug that provides efficacious therapy and is safe (without serious side effects)

Answer 35

THERAPEUTIC WINDOW

Question 36

The process in which the therapeutic agent is combined with different substances to produce a final medicinal
product (e.g., a tablet, infusion solution, etc.). Formulation optimization is ongoing throughout pre-clinical and clinical stages. It ensures drugs are absorbed into the body and delivered to the proper organ at the right time and the right amount

Answer 36

DRUG FORMULATION

Question 37

exclusive right granted by the governments for an invention. Patents give an inventor (academic group
or a private company) the exclusive right to prevent others from making, using, selling, or importing a product or
process based on the patented invention without the inventor’s prior permission, such as through a patent license.
Patent protection is limited to the country or region where it was issued and limited in time, typically 20 years from the date of patent application filing. Pharmaceutical patents can be extended for new indications or novel formulations.

Answer 37

PATENT