3. BIOTECHNOLOGY
⢠It is the area of biology that uses living processes,
organisms or systems to manufacture products or
technology intended to improve the quality of human life.
Depending on the technology, tools and applications
involved, biotechnology can overlap with molecular
biology, bionics, bioengineering, genetic
engineering and nanotechnology.
4. HISTORY
⢠People have been harnessing biological processes to improve their quality of
life for some 10,000 years, beginning with the first agricultural communities.
Approximately 6,000 years ago, humans began to tap the biological processes
of microorganisms in order to make bread, alcoholic beverages, and cheese and
to preserve dairy products. But such processes are not what is meant today
by biotechnology, a term first widely applied to the molecular and cellular
technologies that began to emerge in the 1960s and â70s. A fledgling
âbiotechâ industry began to coalesce in the mid- to late 1970s, led
by Genentech, a pharmaceutical company established in 1976 by Robert A.
Swanson and Herbert W. Boyer to commercialize the recombinant DNA
technology pioneered by Boyer, Paul Berg, and Stanley N. Cohen. Early
companies such as Genentech, Amgen, Biogen, Cetus, and Genex began
by manufacturing genetically engineered substances primarily for medical and
environmental uses.
5. BIOTECHNOLOGY AND PHARMACEUTICAL
INDUSTRY
⢠Pharmaceutical biotechnology is a relatively new
and growing field in which the principles of
biotechnology are applied to the development of
drugs. A majority of therapeutic drugs in the
current market are bio formulations , such as
antibodies, nucleic acid products and vaccines.
7. APPLICATIONS IN PHARMACY
⢠Biotechnological methods have become an
important tool in pharmaceutical drug research
and development . The most relevant indications
are oncology, metabolic disorders and disorders of
the musculoskeletal system
8.
9. APPLICATION IN PHARMACY
EXAMPLES:
⢠INSULIN:
An hormone used diabetes mellitus
⢠GENE THERAPY:
The common function of gene therapy involves the use of functional DNA to
replace muted DNA
⢠CLOTTING FACTORS:
Use in treatment of hemophilia where there is absence of clotting factors in
patientâs body
⢠HUMAN SERUM ALBUMIN:
One of the most blood protein use in the treatment of burns
⢠ENGINEERED ENZYME:
Used to treat variety of conditions that is due to enzyme deficiency
11. genetics
⢠Genetics is the study and science of heredity.
⢠Heredity is a biological process where a parent
passes certain genes and genetic trait onto their
children or offspring.
⢠Genetics is controlled by the chromosomes in the
nucleus of cells
⢠Chromosomes are composed of smaller units called
genes.
⢠Human beings have 23 pairs of chromosomes.
⢠1-22 pairs of chromosomes are called Autosomes.
⢠Pair no. 23rd are the Sex chromosomes.
14. Mendel's laws of inheritance
⢠Mendel's studies yielded three "laws" of
inheritance:
⢠The law of dominance
⢠The law of segregation
⢠The law of independent assortment.
Mendel's Law of Dominance:
⢠It states that:
âIn a cross of parents that are pure for
contrasting traits, only one form of the trait will
appear in the next generation. Offspring that are
hybrid for a trait will have only the dominant trait
in the phenotype.â
Punnett diagram:
⢠It determines the possible genotype and
phenotype of the particular cross
15. genomics
⢠THE Genetic Book Of Life In humans genetic information, also known as our genome, can be
described as the âBook of Lifeâ.
⢠It is the study of all genes present in an organism.
⢠Genomics aims to understand the structure of the genome, including the mapping genes
and sequencing the DNA.
⢠Genomics examines the molecular mechanisms and the interplay of genetic and
environmental factors in disease.
⢠It is useful in the field of medicine and early detection and diagnosis of the genetic diseases
and also in the field of agriculture.
Genetics vs. genomics:
16. Steps of genome sequencing
⢠The shotgun phase of the Human Genome Project itself
consisted of three steps:
1. Obtaining a DNA clone to sequence by the centrifugation
process.
2. Sequencing the DNA clone.
3. Assembling sequence data from multiple clones to determine
overlap and establish a contiguous sequence.
17. proteomics
⢠Proteomics is the large-scale study of proteomes.
⢠A proteome is a set of proteins produced in an organism, system, or biological context.
⢠The proteome is not constant; it differs from cell to cell and changes over time.
⢠Protein activity is also modulated by many factors in addition to the expression level of the relevant gene.
Biological Uses:
⢠Proteomics is used to investigate:
⢠When and where proteins are expressed
⢠Rates of protein production, degradation, and steady-state abundance
⢠How proteins are modified
⢠The movement of proteins between sub-cellular compartments
⢠The involvement of proteins in metabolic pathways
⢠How proteins interact with one another
19. DRUG TARGET
⢠The term "biological target" is frequently used in pharmceutical research to
describe the native protein in the body whose activity is modified by a drug
resulting in a specific effect, which may be a desirable therapeutic effect or an
unwanted adverse effect. In this context, the biological target is often referred
to as a drug target.
⢠Common drug targets include:
⢠G protein (target of 50% drugs)
⢠Enzymes
⢠Ion channels
⢠Nuclear hormonal receptors
⢠Structural proteins like tubulin
⢠Membrane transport proteins
⢠Nucleic acid
20. BIOMOLECULAR TARGET IDENTIFICATION
⢠Target identification and characterization begins with
identifying the function of a possible therapeutic target
(gene/protein) and its role in the disease. Identification of
the target is followed by characterization of the molecular
mechanisms addressed by the target. A good target should
be efficacious, safe, meet clinical and commercial
requirements and be âdruggable
21. CHARACTERSTICS OF A GOOD TARGET
⢠Drug target is a bio molecule normally a protein that could exist in
isolated and complex modality.
⢠The bio molecule have special sites that match other molecules. These
can be endogenous or extrenous substances that could be drugs.
⢠The bio molecule might change structure when the bio molecule binds
to small molecules and changes in structure are normally reversible.
⢠The structure, the activity and expression of the bio molecule might
change over the duration of pathological process.
⢠Small molecules binding to bio molecules are drugs.
22. PROPERTIES OF A GOOD TARGET
⢠The target has a confirmed role in the pathophysiology of a
disease and/or is disease-modifying.
⢠Target expression is not evenly distributed throughout the
body.
⢠The targetâs 3D-structure is available to assess druggability.
⢠The target is easily âassayableâ enabling high through put
screening.
⢠The target possesses a promising toxicity profile, potential
adverse effects can be predicted using phenotypic data
23. TOOLS FOR TARGET IDENTIFICATION
⢠Disease association (genetics and expression changes)
⢠Bioactive molecules
⢠Cell based models
⢠Protein interactions (pull-down assays, yeast 2 hybrid)
⢠Analysis of signaling pathways
⢠Functional analysis (overexpression, transgenics, antisense RNA, gene
variants)
25. PHARMACOGENOMICS
⢠Pharmacogenomics is the study of the role of the genome in drug
response.
⢠Its name (pharmaco + genomics) reflects its combining of
pharmacology and genomics. Pharmacogenomics analyzes how
the genetic makeup of an individual affects his/her response to
drugs.
⢠It deals with the influence of acquired and inherited genetic
variation on drug response in patients by correlating gene
expression or single-nucleotide polymorphisms with
pharmacokinetics (drug absorption, distribution, metabolism, and
elimination) and pharmacodynamics (effects mediated through a
drug's biological targets)
26. ⢠The term pharmacogenomics is often used interchangeably with
pharmacogenetics. Although both terms relate to drug response based on
genetic influences, pharmacogenetics focuses on single drug-gene interactions,
while pharmacogenomics encompasses a more genome-wide association
approach, incorporating genomics and epigenetics while dealing with the effects
of multiple genes on drug response.
27. AIMS OF PHARMACOGENOMICS
⢠Pharmacogenomics aims to develop rational means to
optimize drug therapy, with respect to the patientâs
genotype, to ensure maximum efficiency with minimal
adverse effects.
⢠Through the utilization of pharmacogenomics, it is hoped
that pharmaceutical drug treatments can deviate from what
is dubbed as the "one-dose-fits-all" approach.
⢠Pharmacogenomics also attempts to eliminate the trial-and-
error method of prescribing, allowing physicians to take into
consideration their patient's genes, the functionality of these
genes, and how this may affect the efficacy of the patient's
current or future treatments (and where applicable, provide
an explanation for the failure of past treatments).
28. IMPORTANCE OF PHARMACOGENOMICS
⢠Few more commonly known applications of
pharmacogenomics:
⢠Improve drug safety, and reduce ADRs
⢠Tailor treatments to meet patientâs unique genetic pre-
disposition, identifying optimal dosing
⢠Improve drug discovery targeted to human disease
⢠Improve proof of principle for efficacy trials.
⢠Pharmacogenomics may be applied to several areas of
medicine, including pain management, cardiology, oncology,
and psychiatry
30. GENE THERAPY
⢠An experimental technique for
correcting defective genes that
are responsible for disease
development.
⢠The most common form of gene
therapy involves inserting a
normal gene to replace an
abnormal gene.
31. TYPES OF GENE THERAPY
SOMATIC CELLS
⢠Therapeutic genes transferred into the
somatic cells.
⢠E.g. Introduction of genes into bone
marrow cells, blood cells, skin cells etc.
⢠Will not be inherited later generations.
(Non reproductive)
GERM LINE CELLS
⢠Therapeutic genes transferred into the
germ cells.
⢠E.g. Genes introduced into eggs and
sperms.
⢠It is heritable and passed on to later
generations.
33. ADVANTAGES
⢠Gene therapy has the potential to
eliminate and prevent hereditary
diseases such as cystic fibrosis, ADA-
SCID etc.
⢠It is a possible cure for heart disease,
AIDS and cancer.
⢠It gives someone born with a genetic
disease a chance to life.
⢠It can be used to eradicate diseases from
the future generations.
DISADVANTAGES
⢠Long lasting therapy is not achieved by
gene therapy; Due to rapid dividing of
cells benefits of gene therapy is short
lived.
⢠Immune response to the transferred
gene stimulates a potential risk to gene
therapy.
⢠Viruses used as vectors for gene transfer
may cause toxicity, immune responses,
and inflammatory reactions in the host.
⢠Disorders caused by defects in multiple
genes cannot be treated effectively using
gene therapy
34. MEDICAL USES OF GENE THERAPY
⢠Replace missing or defective genes
⢠Deliver gene that speed up the destruction of cancer cells
⢠Supply gene that cause cancer cells to revert back to normal cells
⢠Deliver bacterial or viral genes as a form of vaccination
⢠Provide genes that promote the growth of new tissues
⢠Deliver genes that stimulate the healing of damaged tissue
37. NUCLEIC ACID THERAPEUTICS
⢠A nucleic acid is a complex , high molecules
weight biochemical macromolecules which is
composed of nucleotides chain that transfer
genetic information .
⢠Nucleic acid molecules are useful in a variety of
biochemical , diagnostic & therapeutics
application.
⢠Gene therapy is a technique used to correct
defective gene which are responsible for disease
development.
⢠Gene therapy involves the transference of new
genetic material to the cell for obtaining a
therapeutic benefits .
38. NUCEIC ACID THERAPEUTICS
⢠Nucleic acid-based molecules (deoxyribonucleic acid, complementary
deoxyribonucleic acid, complete genes, ribonucleic acid, and
oligonucleotides) are utilized as research tools within the broad
borders of gene therapy and the emerging field of molecular medicine
⢠Although most of the nucleic acid-based drugs are in early stages of
clinical trials, these classes of compounds have emerged in recent years
to yield extremely promising candidates for drug therapy to a wide
range of diseases, including cancer, infectious diseases, diabetes,
cardiovascular, inflammatory, and neurodegenerative diseases, cystic
fibrosis, hemophilia, and other genetic disorders.
40. Pharmaceutical importance of nucleic acid therapeutics
⢠Used in cancer, cardiovascular and autoimmune
diseases.
⢠Control gene regulation, transcription , translation
and replication.
