W15 Biologics Flashcards
what do biopharmaceuticals include
proteins
antibodies + oligonucleotides
what are first generation biopharmaceuticals
mainly copies of endogenous proteins or antibodies produced by recombinant DNA technology
what is second generation biopharmaceuticals
engineered to improve the performance of the protein or antibody
What is recombinant DNA
plasmid taken and it is inserted contains cDNA version of mRNA require
What factors do we take into account when trying to harvest a protein
potency
specificity
side effects
delivery
what delivery factors need to be considered when choosing a protein to harvest
cant be delivered orally especially if peptide or protein
cant cross the blood brain barrier *may even require surgery
broken down by ubiquitous proteases
what other factors do we need to take into account when choosing a protein to harvest (manufacturing)
Costs more to make than orgnaic chemicals
Yields may be low
Purification
Quality control
what other factors do we need to take into account when choosing a protein to harvest (stability)
proteins often unstable
sensitive to heat, light etc
storage/ shelf life an issue
what are the main areas for investment when harvesting a protein
formulation
production
what is insulin used for
replaces insulin in type 1 diabetics who have reduced ability to produce insulin
What is somatotropin used for
growth hormone to prevent reduced stature
what is factor 8 used for
a coagulation factor for haemophilia replaces a missing clotting factor in the blood.
what is insulin mechanism of action
insulin receptor agonist
how does the mechanism of insulin work
increases glucose uptake
decreases glucose formation
increases glucose use
how is insulin administered
subcutaneous injections, are usually the most convenient way for patients to self administer a dose
how are long acting form of insulin modified
a modified to increase half life so it is longer acting
what does erythropoetin do
EPO stimulates the production of red blood cells in treatment of chronic renal failure and anaemia associated with EPO deficiency
what does platelet derived growth factor do
wound healing
what is bone morphogenic proteins used for
BMP2 is used for orthopaedic surgery which promotes bone repair
BMP7 promotes vertebral fusion
what is interferons used for
interferon alpha used for hepatitis
interferon beta used multiple sclerosis
what converts plasminogen into plasmin
tissue plasminogen activator *tPA
what is tPA is a serine protease enzyme acts as
clotbuster
when is tPA administered
within 3 hours of stroke onset improves clinical out come at 3 months
what is a monoclonal antibody
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.
what do monoclonal antibody bind to
natural ligands where they neutralise effect
what are monoclonal antibodies also known as
disease modifying agents
What are nanobodies used for
nanobodies are used to develop treatments for diffucult targets such as ion channels
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
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
what are two forms of origin for genetic disease
single gene mutation
multiple gene component
what are two forms of gene therapy
germline
Somatic cell
what is germline gene therapy
starts at a germline using sperm and egg cells however large ethical concerns
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.
what is the route of delivery
targeting system
gene regulatory elements
successful genetic modification
