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Therapeutics > W15 Biologics > Flashcards

W15 Biologics Flashcards

1
Q

what do biopharmaceuticals include

A

proteins

antibodies + oligonucleotides

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2
Q

what are first generation biopharmaceuticals

A

mainly copies of endogenous proteins or antibodies produced by recombinant DNA technology

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3
Q

what is second generation biopharmaceuticals

A

engineered to improve the performance of the protein or antibody

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4
Q

What is recombinant DNA

A

plasmid taken and it is inserted contains cDNA version of mRNA require

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5
Q

What factors do we take into account when trying to harvest a protein

A

potency
specificity
side effects
delivery

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6
Q

what delivery factors need to be considered when choosing a protein to harvest

A

cant be delivered orally especially if peptide or protein

cant cross the blood brain barrier *may even require surgery

broken down by ubiquitous proteases

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7
Q

what other factors do we need to take into account when choosing a protein to harvest (manufacturing)

A

Costs more to make than orgnaic chemicals

Yields may be low

Purification

Quality control

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8
Q

what other factors do we need to take into account when choosing a protein to harvest (stability)

A

proteins often unstable

sensitive to heat, light etc

storage/ shelf life an issue

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9
Q

what are the main areas for investment when harvesting a protein

A

formulation

production

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10
Q

what is insulin used for

A

replaces insulin in type 1 diabetics who have reduced ability to produce insulin

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11
Q

What is somatotropin used for

A

growth hormone to prevent reduced stature

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12
Q

what is factor 8 used for

A

a coagulation factor for haemophilia replaces a missing clotting factor in the blood.

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13
Q

what is insulin mechanism of action

A

insulin receptor agonist

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14
Q

how does the mechanism of insulin work

A

increases glucose uptake
decreases glucose formation
increases glucose use

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15
Q

how is insulin administered

A

subcutaneous injections, are usually the most convenient way for patients to self administer a dose

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16
Q

how are long acting form of insulin modified

A

a modified to increase half life so it is longer acting

17
Q

what does erythropoetin do

A

EPO stimulates the production of red blood cells in treatment of chronic renal failure and anaemia associated with EPO deficiency

18
Q

what does platelet derived growth factor do

A

wound healing

19
Q

what is bone morphogenic proteins used for

A

BMP2 is used for orthopaedic surgery which promotes bone repair

BMP7 promotes vertebral fusion

20
Q

what is interferons used for

A

interferon alpha used for hepatitis

interferon beta used multiple sclerosis

21
Q

what converts plasminogen into plasmin

A

tissue plasminogen activator *tPA

22
Q

what is tPA is a serine protease enzyme acts as

A

clotbuster

23
Q

when is tPA administered

A

within 3 hours of stroke onset improves clinical out come at 3 months

24
Q

what is a monoclonal antibody

A

Monoclonal antibodies are laboratory-produced molecules engineered to serve as substitute antibodies that can restore, enhance, modify or mimic the immune system’s attack on cells that aren’t wanted, such as cancer cells.

25
Q

what do monoclonal antibody bind to

A

natural ligands where they neutralise effect

26
Q

what are monoclonal antibodies also known as

A

disease modifying agents

27
Q

What are nanobodies used for

A

nanobodies are used to develop treatments for diffucult targets such as ion channels

28
Q

what is gene therapy

A

replacement of a defective genes with a normal healthy genes to alleviate or cure disease

represents an opportunity for the treatment of genetic disorders in adults and children by genetic modification of human body cells

29
Q

what are potential applications

A

radical cure of monogenic disease

amelioration of disease with or without a genetic component including many malignant, neurodegenrative and infectious diseases

30
Q

what are two forms of origin for genetic disease

A

single gene mutation

multiple gene component

31
Q

what are two forms of gene therapy

A

germline

Somatic cell

32
Q

what is germline gene therapy

A

starts at a germline using sperm and egg cells however large ethical concerns

33
Q

what is somatic cell gene therapy

A

targeting only cells that live as long as the individual, make up all approved gene therapy trails in humans.

34
Q

what is the route of delivery

A

targeting system

gene regulatory elements

successful genetic modification

Therapeutics (50 decks)

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