Introduction to Biotechnology

What is biotechnology?

Any technological applciation that uses biological systems, living organisms, or parts of living organisms to make/modify those living things for a specific use

How does the biotech industry work?

Large companies: profits from sales of existing products
Small companies and startups: venture capital and government grants
Academic institutions: government grants, endowments

What are the major events in the history of biotechnology?

4000 BC: Egyptians used yeast to make break
1700s: cross breed different plants (Native Americans)
1797: Jenner created smallpox vaccine
1865: Mendel, genetics, pea plants (Carver with peanuts)
1919: first use of word biotechnology
1981: FDA approved first recombinant DNA biotech drugL penicillin created in genetically modified bacteria

Pharmaceutical vs biotech companies

Pharma: large, traditional, chemical expertise, drug delivery, chemical synthesis, private investors

Biotech: small, innovative, biological expertise, drug discovery, biological processes, private investors

Type 1 vs Type 2 Diabetes

Type 1: insulin dependent - born with a mutation that prevents pancreas from making insulin (receptor never activated and glucose unable to enter cell)

Type 2: body can make insulin but cells stop responding to it (receptor is not sensitive to insulin anymore)

Biotech to treat diabetes patients

The plasmid of DNA is removed

gene for human insulin from a non-diabetic patient is inserted into bacterial plasmid

new plasmid is re-inserted back into bacteria and allowed to reproduce

insulin is harvested and purified from bacteria - medicine (Humulin!)

Sharing work

Peer reviewed publications, conferences, meetings, seminars, posters, patents

Steps from concept to commercial drug product

Agriculture: trait generation, trait optimization, proof of concept in model plant, early testing/development, field trials, commercialization

Biopharamceutical: trait identification/validation, lead generation, lead optimization, preclinical research, clinical trials, commercialization

Biotech reguluations

US Department of Agri (plants with potential plant-pest risks)

US EPA (pesticides)

US Food and Drug Agency (crops for food and feed)

Good laboratory practice

quality system with a set of reguluated practices used to collect safety data on a drug or product being developed

Good manufacturing practice

quality system with a set of standardized practice that ensures steps are being taken to produce consistently safe and affective products

HIV

Human immunodeficiency virus

• replicates inside living cells - HIV replicates CD4 T-cells (humans only)

• releases RNA and proteins into cytoplasm - converts RNA to DNA

• HIV DNA integrates with host DNA, transcribed by host enzymes

• new viruses are assembled at cell surface

• new viruses leave the cell, budding off from host

• kills immune cells

AIDS

acquired immunodeficiency syndrome

• opportunistic infections: fungal mengingitis, kaposi's sarcoma, pneumocystis pneumonia

• HIV spread: blood, sexual interaction, from mother to child during childbirth and breastfeeding

Why do we need HIV drugs?

• drugs target specific proteins involved in HIV replication

• main receptor is CD4 (helps immune system recognize pathogens and HIV takes advantage of this)

• HIV drugs are extremely toxic (patients stop taking them)

• HIV mutates to become resistant to treatments (patients take a cocktail to target many proteins on virus)

Virologic failure

when the drug treatments stop keeping the HIV virus in check

Stages of clinical trial

pre-clinical: tests if treatment is safe, best way to give treatment, dosage - cells and animals

phase II trial: second stage of testing a new drug in healthy humans

phase III: toxicity and efficacy in HIV-infected humans

phase IV: long term benefits and side effects

What is AZT

Zidovudine - type of anti-HIV drug to treat (not cure) the disease

- used to prevent mother-child spread and needle exposure

Toxicity

how much damage a drug causes the body (how many side effects and how serious those side effects are)

Efficacy

how well the drug works (how well it lowers the amount of HIV in the body)

Standard of care

the most common treatmente for a particular disease that also best fits the patient's needs

Half life

time for the plasma concentration of a drug to reduce to half its original value
- how long it takes for a drug to be removed from your body

How does patient become resistant to HIV drugs?

patient stops taking the drug and mutations occur which the drug cannot fight against anymore