Biotechnology & Drug Discovery.

Presented by:
Irsa Jamal
Drug discovery is the process through which potential
new medicines are identified. It involves a wide range
of scientific disciplines, including biology, chemistry
and pharmacology.
An expensive process due to the high costs of R&D

and human clinical tests.
The average total cost per drug development varies
from US$ 897 million to US$ 1.9 billion.
The typical development time is 10-15 years.
Ways of Drug Discovery
Accidental Discoveries Some drugs have been discovered by accident. (For example
Warfarin (Coumadine) was first used as a rat poison. Rats
have died of bleeding. Now, it is used as per oral
anticoagulant.)
Natural Substances Herbal medicaments and other substances that could be

derived from plants. (For example digitalis) or from fungi or
other natural sources.
Computer Technology - Perform structure/activity/function computations of
Molecular Structuring computer-readable chemical libraries with a huge number of
(Molecular Docking) chemical compounds.
Direct Research Creation of chemical structures that are likely to solve

medical problems. First, there is a problem that should be
solved. The researchers are looking for a pharmaceutical
solution.
Changing of known The aim is to improve the existing medicaments. To
Molecular Compounds eliminate side effects. To change the way of drug delivery
(Per oral P.O., intramuscularly I.M., intravenous I.V. or
other).
Genetic Engineering For example, in order to avoid undesirable allergic reactions
(Bio Technology) to insulin produced from pigs, a human insulin gene was
genetically included into one cell organisms that started to
produce human insulin
Biopharmaceutical Drug - A medical drug
derived from biological sources and
produced by biotechnology.
 The primary difference between

biopharmaceuticals and traditional
pharmaceuticals is the method by which the drugs
are produced:
 Biopharmaceutical : manufactured in living
organisms such as bacteria, yeast and mammalian
cells,
 Traditional Pharmaceutical: manufactured through
a series of chemical synthesis.
 Although it seems like a
new thing, biotechnology
has actually been around for
a while:
◦ Domesticated plants and

animals are the result of
selective breeding
◦ Using yeast to make bread rise
◦ Using bacteria or yeast to
ferment grapes into wine
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 uses
Biotech is the term used for biotechnology or
products produced by biotechnology. These
drugs are also called biologicals, biotech
drugs, biological drugs, or
biopharmaceuticals.
THERAPEUTIC
PROTEINS
TISSUES AND MONOCLONAL

CELLS ANTIBODIES
BIOTECH
BLOOD
VACCINES
COMPONENTS
IMMUNO-
THERAPY
 Also called red biotechnology
 It includes:
o Production of medicines and pharmaceutical
products for treating or diagnosing disorders
o Designing of organisms to manufacture
antibiotics and vaccines
o Engineering of genetic defects through
genomic manipulation
o Use in forensics through DNA profiling
 Production of human insulin from non-
human sources.
 Production of hormones like Interferons,

Cytokinins, Steroids and human growth
hormones.
 Gene therapy for prevention and control of

diseases like hemophilia cystic fibrosis
 Development of vaccines and antibodies for

rabies, HIV, etc.
 Drug production
 Pharmacogenomics
 Gene therapy
 Producing medicines through:
 Isolating enzymes
 Genetically engineering enzymes

 Recently, plants are being genetically
modified to produce pharmaceutical products
instead of their natural compounds
 For Example:
A drug Elelyso for treating Gaucher is being
produced by genetically engineering carrots
 INSULIN:
Human insulin is being produced using genetic

engineering technique known as humulin and it
is used for the treatment of diabetes that is low
sugar level in the blood…..
 INTERFERON:
o Interferon interfere in transmission of viral genome

from one cell to another and it also inhibits the cell
division of abnormal cells.
o Interferon produced using the recombinant DNA

technology is used to treat cancer patients.
o Interferon improved the quality of life of cancer

patients…..
 HUMAN GROWTH HORMONE:
Since dwarfism is caused by growth hormone

deficiency so it can be diagnose by HGH
testing.
So HGH is used for the treatment of dwarfism
due to hypo pituitary activity.
 Pharma = Drug or Medicine
 Genomics = The study of genes
 Studying response of genetic make up
of an individual to a drug or
pharmaceutical products
 “One-size-fits-all drugs” only work for about 60
percent of the population at best. And the other 40
percent of the population increase their risks
of adverse drug reaction because their genes do not do
what is intended of them.
 Helps in the development of tailor made medicines
 Ensures more appropriate methods of

determining drug dosages
 Improve process of drug discovery and approval
 Obtaining of better and safer vaccination
 Decrease in the overall cost of Health Care
 Advanced Screening for Disease

 Herceptin
 Gleevec
 Erbitux
 Tumoricide
Some barriers faced are:
 Complexity of finding gene variation that

affect drug response
 Limited drug alternatives
 Disincentives for drug companies to make

multiple pharmacogenomic products
 Educating healthcare providers

 The process in which a faulty gene is
removed or replaced with its healthy copy to
restore the normal function of that gene
 Replacing a mutated gene that causes disease
with a healthy copy of the gene
 Inactivating or “knocking out” a mutated gene

that is functioning improperly
 Introducing the new gene that help fight a

disease
 Some common ways are:
 Using fat droplets in nose sprays
 Using cold viruses that are modified to carry

alleles ,go into the cell and affect them
 The direct injection of DNA(might include

electroporation or biolistic method)
The process of gene therapy is of two types:
 Stem cell gene therapy:

In this gene therapy is applied on a fully developed
organism and the effects of gene therapy lasts only
to the operated organism
 Germ line gene therapy:

In this process gene therapy is done on a fertilized
egg or an early embryo and the altered genome is
followed in next generations.
The following are methods for finding a drug
candidate,
virtual screening, Computational methods which is
used to predict or simulate how a particular compound
interacts with a given protein target
The real screening, such as high-throughput

screening (HTS), can experimentally test the activity of
hundreds of thousands of compounds against the
target a day.
 based on the computationally inferred or simulated
real screening;
The main advantages of this method compared to
laboratory experiments are:
o low costs, no compounds have to be purchased
externally or synthesized by a chemist;
o it is possible to investigate compounds that have not
been synthesized yet;
o conducting HTS experiments is expensive and VS can
be used to reduce the initial number of compounds
before using HTS methods;
 A method which predicts the preferred orientation of one molecule
to a second when bound to each other to form a stable complex
 When the structure of the target is available, usually from X-ray

crystallography, the most commonly used virtual screening method is
molecular docking.
 It can also be used to test possible hypotheses before conducting

costly laboratory experiments.
 Molecular docking programs predict how a drug candidate binds to

a protein target
 Metaphorically, the active site of the protein can be viewed as a

lock, and the ligand can be thought of as a key. Molecular docking is
the process of testing whether a given key fits a particular lock
The software consists of two core components:
A search algorithm
◦ responsible for finding the best conformations of the
ligand,(a small drug-like molecule and protein system)
◦ A conformation is the position and orientation of the

ligand relative to the protein
◦ The conformation also contains information about the

internal flexible structure of the ligand and in some cases
about the internal flexible structure of the protein.
Since the number of possible conformations is extremely
large, it is not possible to test all of them
An evaluation function, sometimes called a score function.
This is a function providing a measure of how strongly a
given ligand will interact with a particular protein
Energy force fields (often used as evaluation functions.)

calculate the energy contribution from different terms such
as
the known electrostatic forces between the atoms in the
ligand and in the protein
forces arising from deformation of the ligand
pure electron-shell repulsion between atoms
effect from the solvent in which the interaction takes
place.
 http://www.combichemistry.com/drug-
discovery.html
 http://www.intechopen.com/books/protein-
engineering-technology-and-
application/protein-protein-and-protein-
ligand-docking

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Presented by:

An expensive process due to the high costs of R&D

Natural Substances Herbal medicaments and other substances that could be

Direct Research Creation of chemical structures that are likely to solve

 The primary difference between

◦ Domesticated plants and

TISSUES AND MONOCLONAL

 Production of hormones like Interferons,

 Gene therapy for prevention and control of

 Development of vaccines and antibodies for

 Genetically engineering enzymes

Human insulin is being produced using genetic

o Interferon interfere in transmission of viral genome

o Interferon produced using the recombinant DNA

o Interferon improved the quality of life of cancer

Since dwarfism is caused by growth hormone

 Ensures more appropriate methods of

 Improve process of drug discovery and approval

 Obtaining of better and safer vaccination

 Decrease in the overall cost of Health Care

 Advanced Screening for Disease

 Complexity of finding gene variation that

 Limited drug alternatives

 Disincentives for drug companies to make

 Educating healthcare providers

 Inactivating or “knocking out” a mutated gene

 Introducing the new gene that help fight a

 Using fat droplets in nose sprays

 Using cold viruses that are modified to carry

 The direct injection of DNA(might include

 Stem cell gene therapy:

 Germ line gene therapy:

The real screening, such as high-throughput

 When the structure of the target is available, usually from X-ray

 It can also be used to test possible hypotheses before conducting

 Molecular docking programs predict how a drug candidate binds to

 Metaphorically, the active site of the protein can be viewed as a

◦ A conformation is the position and orientation of the

◦ The conformation also contains information about the

Energy force fields (often used as evaluation functions.)

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