Biopharmaceuticals and gene therapy

Question 1

What are biopharmaceuticals?

Answer 1

A biological molecule used as a pharmaceutical.

Question 2

What is a first generation biopharmaceutical?

Answer 2

Biological molecules which haven’t been modified in any way. (IE: Human Growth Hormone).

Question 3

What is a second generation biopharmaceutical?

Answer 3

Biological molecule which undergoes modification and alters the nature of the product.

Question 4

What are the production methods for biopharmaceuticals?

Answer 4

They have a bacterial expression system - the human gene is combined with the bacterial plasmid to create a recombinant plasmid.
Bacterial transformation occurs and therefore, heterogenous expression occurs.
Therefore, protein purification occurs too.

Question 5

What is the advantage of the production method?

Answer 5

A high yield is expressed.

Question 6

What is the disadvantage of the production method?

Answer 6

May contain endotoxins.

Post translational processing occurs which can differ compared to mammalian cells.

Question 7

What are the alternative expression systems?

Answer 7

Chinese hamster ovary cells.
Plants: Tobacco / Edible plants.
Transgenic cattle.

Question 8

What is an issue of producing purer products?

Answer 8

It is more costly.

Question 9

What are the advantages of using plants / transgenic cattle for alternative expression systems?

Answer 9

Genes can be put into tobacco easier = so can be done quicker and more efficiently.
Transgenic cattle can produce milk which has proteins which can utilise the production of proteins.

Question 10

What are the problems with first generation mAB’s?

Answer 10

It can provoke an immune response
Furthermore, has a short half life.
It also can’t activate human complements.

Question 11

What are some of the solutions to dealing with the failure of activation of human complements?

Answer 11

Humanising the antibodies - chimera is formed.
This chimera is a mix between human and mice.
This results in specificity and therefore, effective therapeutics can be generated.

Question 12

What is an alternative to delivering entire genes?

Answer 12

Usage of short antisense oligonucleotides.

Question 13

What is the size difference between biopharmaceuticals and conventional drugs?

Answer 13

Biopharmaceuticals tend to be larger compared to conventional drugs.

Question 14

What is the synthesis between biopharmaceuticals and conventional drugs?

Answer 14

Easy to synthesise identical batches when using a conventional drug.

Question 15

What is the relationship between dose and effect?

Answer 15

In conventional drugs there is usually predictable relationship between dose and effect.
Whereas with biopharmaceutical drugs, there are complex mechanisms.

Question 16

Why are biopharmaceuticals harder to synthesise?

Answer 16

It is a harder process.

Question 17

What is gene therapy?

Answer 17

It is a strategy which delivers a functioning copy of a defective gene to a target cell using a suitable vector.

Question 18

What are the therapeutic applications?

Answer 18

Gendicine = Treatment for replacing faulty p53 protein. 
Glybera = Delivers correct copy of lipoprotein lipase to patients suffering from lipoprotein lipase deficiency.  
Strimvelis = Gene therapy treats SCID due to deaminase deficiency.