Pharmaceutical

Biotechnology
CCC-409

Tushar Kapur
Ranbir Negi
Samarth Agarwal
Prakhar Dubey
 What is Pharmaceutical Biotechnology?

Can simply be defined as the science that covers all technologies required for the
production, manufacturing and registration of biological drugs.

Pharmaceutical biotechnology consists of the combination of two branches which

are “pharmaceutical science” and “biotechnology”

Pharmaceutical Science: Can simply be defined as the branch of science that deals
with the formulation compounding and dispensing of drugs.

Biotechnology: can simply be defined as the application of biological system, living

organisms, or their derivatives in making or modifying products or processes for
specific use.
 Aim

The aim of pharmaceutical biotechnology is to design, produce drugs that are

adapted to each person's genetic makeup, which can give the maximum
therapeutic effect. Biotechnology plays an important role in pharmaceutical
science most especially in the pharmaceutical industries by creation of genetically
modified organisms that can be used in industrial production.

It is a relatively new and growing field which contributes to the design and delivery
of new therapeutic drugs, the development of diagnostic agents for medical tests,
and the beginnings of gene therapy for correcting the medical symptoms of
hereditary diseases.
 History of the pharmaceutical industry

At the turn of the twentieth century, the medical community had at their disposal
only four drugs that were effective in treating specific diseases:

Digitalis: Used to treat various heart conditions

Quinine: Used to treat malaria

Ipecacuanha: Used for treating dysentery

Mercury: For the treatment of syphilis

 History of the pharmaceutical industry (contd.)
Developments in biology (particularly the growing realization of the microbiological
basis of many diseases), as well as a developing appreciation of the principles of
organic chemistry, helped underpin future innovation in the fledgling
pharmaceutical industry.

The discovery and chemical synthesis of the sulfa drugs for the treatment of
bacterial infections was a landmark discovery.

Although the pharmaceutical potential of these regulatory molecules was generally

appreciated, their widespread medical application was in most cases rendered
impractical due to the tiny quantities in which they were naturally produced. This
created a lack of appropriate, safe and effective medicine.
 The advent of biopharmaceuticals
The discovery of recombinant DNA technology (genetic engineering) and monoclonal
antibody technology (hybridoma technology) overcame many such difficulties, and
marked the beginning of a new era of the pharmaceutical sciences:

It overcomes the problem of source availability. Many proteins of therapeutic potential

are produced naturally in the body in minute quantities. This rendered impractical in
their direct extraction from native source material in quantities sufficient to meet likely
clinical demand. Recombinant production allows the manufacturing of any protein in
whatever quantity is required.

(continued in the next slide)

 The advent of biopharmaceuticals (contd.)

It overcomes problems of product safety. Direct extraction of product from some

native biological sources has, in the past, led to the unwitting transmission of
disease. Examples include the transmission of blood-borne pathogens such as
hepatitis B and C and human immunodeficiency virus (HIV) via infected blood
products.

It provides an alternative to direct extraction from inappropriate/dangerous source

material. Ancrod, for example, is a protein displaying anticoagulant activity and is
produced naturally by the Malayan pit viper (by milking the snake venom).
However, recombinant production in less dangerous organisms, such as
Escherichia coli or Saccharomyces cerevisiae, is possible and would be
considered preferable by most.
 Major Classes of Biopharmaceuticals

Extracted from living systems

Produced by recombinant DNA

Vaccines

Gene therapy
Extracted from living systems

Whole blood

Organs and tissue transplant

Stem cell therapy

Passive Immunization
 Recombinant DNA (rDNA) Technology
Recombinant DNA (rDNA) molecules are DNA molecules formed by laboratory
methods of genetic recombination (such as molecular cloning) to bring together
genetic material from multiple sources, creating sequences that would not
otherwise be found in the genome
 Examples of rDNA Biopharmaceuticals

Substances that are (nearly) identical to the body's own key signalling proteins.
Examples are the blood-production stimulating protein erythropoietin or the growth
hormone or biosynthetic human insulin and its analogs.

Monoclonal antibodies: These are similar to the antibodies that the human
immune system uses to fight off bacteria and viruses, but they are "custom-
designed" and can therefore be made specifically to counteract or block any given
substance in the body, or to target any specific cell type.
 Vaccines
A vaccine typically contains an agent that resembles a disease-causing
microorganism and is often made from weakened or killed forms of the microbe,
its toxins, or one of its surface proteins. The agent stimulates the body's to
recognize the agent as a threat, destroy it, and to further recognize and destroy
any of the microorganisms associated with that agent that it may encounter in the
future.
 Gene Therapy

Gene therapy is the therapeutic delivery of DNA or RNA into a patient's cells as a
drug to treat disease.
 Gene Therapy (Contd.)
Types

Somatic: The therapeutic genes are transferred into any cell other than a gamete,
germ cell or an undifferentiated stem cell. Does not affect future generations.

Germline: Germs cells are modified. Affects all future generations.

Delivery Method

Viral: Substituting a virus's genetic material with therapeutic DNA which becomes
a permanent part of the host's DNA in infected cells.

Non viral: Large scale production and low host immunogenicity, but lower
therapeutic efficacy
 Biopharmaceuticals: Future Prospects
In recent years, the biopharmaceutical market has been developing at a faster
rate than the market for all drugs. According to analysts, this market will continue
to grow.

Approximately one in every four new drugs now coming on the market is a
biopharmaceutical.The products include a range of hormones, blood factors and
thrombolytic agents, as well as vaccines and monoclonal antibodies.

The highly promising prospects of the biopharmaceutical market are related to

breakthrough innovations, such as the development of immunotherapy, antibody–
drug conjugates, and gene therapies.

Factors hindering the development of this market may include the high costs of
implementing the developed biopharmaceuticals.
 References

Walsh, Gary. Pharmaceutical biotechnology: concepts and applications. John

Wiley & Sons, 2013.

Kesik‐Brodacka, Malgorzata. "Progress in biopharmaceutical development."

Biotechnology and applied biochemistry 65.3 (2018): 306-322.

https://en.wikipedia.org/wiki/Biopharmaceutical

https://www.omicsonline.org/scholarly/pharmaceutical-biotechnology-journals-
articles-ppts-list.php

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3525971/