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Biotechnology > Quiz 1 > Flashcards

Quiz 1 Flashcards

INtro, biologics, stem cells, big pharma, VC, genome sequencing

1
Q

What is biotechnology?

A

any technique that uses living organisms or substances from those organisms to make or modify a product, to improve plants or animals, or to develop microorganisms for specific use

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2
Q

What is biotransformation?

A

the conversion of a particular organic compound or molecule into a useful product through enzymatic reactions provided by specific microorganisms

ex) microbial production of vanillin

minimizes synthetic chemical steps required for the synthesis of commercially important compounds

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3
Q

What are the advantages of biotransformation?

A
  1. stereospecificity
  2. higher yield
  3. cheaper products
  4. lower use of toxic solvents and reagents
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4
Q

What is a biologic?

A

Any virus, therapeutic serum, toxin, antitoxin, vaccine, blood components or derivatives, cell and tissue-based product, or gene therapy products, which are used in the prevention, treatment, or cure of human beings

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5
Q

Who regulates biologics?

A

branch of FDA called CBER (center for biologics evaluation and research)

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6
Q

What biologics does the FDA regulate?

A
  1. monoclonal antibodies (Herceptin; blocks HER2)
  2. cytokines (immune protein regulators)
  3. therapeutic enzymes
  4. thrombolytic agents (infarction, stroke, embolism–clot busters: tPA)
  5. Hormones
  6. Growth factors
  7. Misc. proteins
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7
Q

What are the advantages of biologics over small molecule therapies?
What are the disadvantages?

A
  1. interact with difficult targets: small molecules cannot break up the PPI
  2. specificity for particular target proteins (antigen + antibody): small molecules can’t differentiate between related PPIs
  3. not oral, more expensive
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8
Q

Examples of biologics?

A

vaccines, insulin, somatotropin (recombinant GH), recombinant erythropoietin for RBCs, etanercept (blocks TNF-alpha for RA), recombinant factor 8 in hemophilia

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9
Q

Enzymes as biologics?

A

industrial: lactase (lactose = glucose + galactose), amylase (starch breakdown), cellulase (hydrocarbons into sugars)

therapeutic: DNAse for CF, botox = cleaves SNARE protein, blocks neurotransmitter release (acetylcholine), paralyzes muscle

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10
Q

What is a biosimilar?

A

patented biologics lose protection and are produced as “generics”

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11
Q

What are the advantages and disadvantages of biosimilars?

A

advantages
- economic savings in development (more efficient manufacturing process, already FDA approved, low risk)

disadvantages
- process differences may result in unpredicted effects (temp, pH, etc.)
- post-translational modifications could influence binding to target, half-life, aggregate formation, immunogenicity

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12
Q

What is cellular differentiation?

A

maturation process of primitive cells into the specialized, functional cell types of the body
blood stem cells: red, white, platelets

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13
Q

What is a stem cell?

A

a single cell that can replicate itself or differentiate into many cell types

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14
Q

What are the 3 properties of SCs?

A
  1. capable of dividing and renewing themselves for long periods
  2. unspecialized
  3. give rise to specialized cell types
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15
Q

Types of stem cells?

A
  1. totipotent: maximum potential; give rise to any type of cell; fertilized egg and cells up to 8 cell stage
  2. pluripotent: potential to make any differentiated cell in the body (ectoderm, endoderm, mesoderm); ES
  3. multipotent: restricted fate to becoming one of a few types of cells; bone marrow
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16
Q

What stage are the majority of embryonic stem cells obtained from?

A

blastocyst: 100-200 cell stage post-fertilization; trophectoderm (placenta) and ICM (fetus)

17
Q

What is a chimera?

A

organism made up of two genetically distinct types of cells from the same or different species (cell or tissue mixture, not genetic mixture = hybrid)

18
Q

How do you isolate ESCs?

A

fertilization, remove ICM, grow in dish, change culture conditions to stimulate cells to differentiate

19
Q

What are the hallmarks of ESCs?

A
  1. express molecular markers: Oct4, Nanog, Sox-2
  2. Immortal: clonal expansion potential, active telomerase
  3. Embryoid Bodies: aggregation of in vitro cells differentiating into structures containing all three germ layers
  4. teratoma formation: cells injected into mice form tumors comprised of tissues representing all three germ layers
20
Q

What are the problems?

A
  1. non-human proteins from the culture media could induce rejection of ES cells when injected in patients
  2. ethical concerns about destroying a human embryo
21
Q

What is somatic cell nuclear transfer (SCNT)?

A

process wherein the genetic material of one cell is replaced with that of another

ES cells generated for therapy (ICM extracted or left (reproductive cloning of donor nucleus parent)

22
Q

What are induced pluripotent stem cells (iPSCs)?

A

a type of pluripotent cell made directly from a somatic cell

23
Q

How are iPSCs made?

A
  1. harvest fibroblasts
  2. infect with Oct4, Sox2, Klf4, and c-Myc viruses
  3. correct mutation in iPS by gene targeting
  4. differentiate into embryoid bodies
  5. transplant corrected version back in
24
Q

What was the concern with iPSCs and how was it fixed?

A

viral insertion near oncogenes or into tumor suppressor genes could cause cancer

  1. inducing genes on plasmids
  2. inducing proteins
  3. inducing mRNAs
25
Q

What drives the creation of large pharma?

A

M&A to enhance drug pipeline and R&D

26
Q

What do Pharma develop?

A
  1. Small molecule drug
    - low molecular weight organic compound, not a polymer
    - not made in living cells
    ex) tetracycline = chemical alteration of antibiotic
  2. Biologics
    - high molecular complexity
    - manufacturing depends on living organisms
27
Q

How to discover a new drug?

A
  1. harnessing or testing novel natural products (Aspirin; NSAID; blocks COX-1)
  2. Identify novel druggable and validated targets and employ large-scale chemical screening methods to discover new chemical compounds (NCEs)
28
Q

What are the effects of differences in chemical composition of drugs?

A
  1. dosage needed to be effective
  2. uptake by different cells and tissues
  3. duration of drug effect
  4. side effects and toxicity
  5. protocols and costs for synthesis, purification, storage, formulation
  6. intellectual property (allowing companies to sell similar drugs)
29
Q

What are the problems with natural products?

A

active agent must be purified, identified, and synthesized

target of action might not be known even if physiological effects are

30
Q

What is pharmacokinetics?

A

what the body does to a drug
- duration, max tolerated concentration, min effective concentration, AUC (drug exposure)
- ADME = (absorption, distribution, metabolism, elimination)

31
Q

what is pharmacodynamics?

A

the effect of a drug on the body

32
Q

What are the clinical trial phases?

A
  1. safety in humans
  2. efficacy at treating disease
  3. larger scale safety and efficacy
  4. long term safety
33
Q

What are the parts of lead optimization?

A

increase potency, improve specificity, reduce metabolism, maximize oral bioavailability, assess protein expression, establish PK/PD, reduce toxicity, mutagenicity

Biotechnology (3 decks)

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