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
what do monoclonal antibody bind to
natural ligands where they neutralise effect
26
what are monoclonal antibodies also known as
disease modifying agents
27
What are nanobodies used for
nanobodies are used to develop treatments for diffucult targets such as ion channels
28
what is gene therapy
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
what are potential applications
radical cure of monogenic disease amelioration of disease with or without a genetic component including many malignant, neurodegenrative and infectious diseases
30
what are two forms of origin for genetic disease
single gene mutation multiple gene component
31
what are two forms of gene therapy
germline | Somatic cell
32
what is germline gene therapy
starts at a germline using sperm and egg cells however large ethical concerns
33
what is somatic cell gene therapy
targeting only cells that live as long as the individual, make up all approved gene therapy trails in humans.
34
what is the route of delivery
targeting system gene regulatory elements successful genetic modification