Drug Development 16.11.23

Question 1

Define druggability

Answer 1

the ability of a protein target to bind small molecules with high affinity (sometimes called ligandability).

Question 2

What are the 5 current models of drug development?

Answer 2

• Lead compound identification
• Pre-clinical research
• Filing for regulatory status
• Clinical trials on humans
• Marketing the drug

Question 3

Name 6 recombinant proteins in clinical use

Answer 3

Insulin
Erythropoietin
Growth hormone
Interleukin 2
Gamma interferom
Interleukin 1 receptor

Question 4

What does gene therapy consist of?

Answer 4

Gene therapy consists of a recombinant nucleic acid used in or administered to a human being with a view to regulating, replacing, adding or deleting a genetic sequence. Its effect relates directly to the recombinant nucleic acid sequence it contains or to the product of genetic expression of this sequence

Question 5

Define rational drug design

Answer 5

The process of finding new medications based on the knowledge of a biological target

Question 6

Rational drug design - general info

Answer 6

The drug is most commonly an organic small molecule that activates or inhibits the function of a biomolecule
Involves the design of molecules that are complementary in shape and charge to the biomolecular target with which they interact and therefore will bind to it
Relies on the knowledge of the 3D structure of the biomolecular target – structure-based drug design