Biologicals and Gene therapy Flashcards
What are biopharmaceuticals
They include protein, antibodies used as drugs
e.g vaccines
Define affinity, potency, efficacy
Affinity
- how well the drug binds to the receptor(lower dose, higher affinity)
potency
- Amount of drug needed to produce therapeutic effect
Efficacy
- ability of a drug-receptor complex to produce a maximum functional response.
Explain FOUR intrinsic challenges in the use of protein therapeutics
Delivery
- Can’t be delivered orally (esp. if peptide/protein)
- Can’t/difficult to cross the blood brain barrier
- May require surgery
- Broken down by ubiquitous proteases
Even if injected rapidly metabolised
Manufacture
-Cost more to make than organic chemicals
- Yields may be low
Stability
- Proteins often unstable as are sensitive to heat, light, etc
- Storage/shelf life is also an issue
Give an example of a protein used in biopharmaceutical, itsindication and mechanism of action
Insulin,
used in type I diabetes mellitus
works as an insulin receptor agonist
What are monoclonal antibodies
- they are very specific antagonists that bind to natural ligand to neutralise the effect
*May contain part of receptor
what is gene therapy
Replacement of a defective gene(s) with a normal, healthy gene(s) to manage or cure disease. e.g in cystic fibrosis
give an example of a disease where monoclonal antibodies are used
Crohn’s disease, cancer, cystic fibrosis
Describe TWO types of gene therapy
Germ-line (sperm/egg)
- Possible permanent cure however ethical concerns such as IQ, hair/eye/ skin colour
Somatic cell (diploid)
- Targeting only cells that live in the patient and not the patients descendants
What are nanobodies
They are used to develop treatments for difficult target.
antibodies are big so hard for them to access their target sites nano-bodies are used for treatment of difficult targets.
Advantage of nanobodies
- theres no accessibility issues because they are smaller
