This document discusses the pharmacokinetics and pharmacodynamics of biotechnology drugs. It begins by defining pharmacokinetics as the study of what the body does to a drug and pharmacodynamics as the study of a drug's biochemical and physiological effects. The document then examines various types of biotechnology products including proteins and peptides, monoclonal antibodies, oligonucleotides, vaccines, and gene therapies. It provides examples and applications of each type of biotechnology drug.

1. Pharmacokinetics And Pharmacodynamics
of Biotechnology Drugs
Dadasaheb Balpande College Of Pharmacy, Besa, Nagpur
Presented By :
Trilok D. Shahare
M.Pharm IInd Sem
Dept. of Pharmaceutics
Guided By :
Dr. V. P. Sable
Dept. of Pharmaceutics
1

2. Contents
 Introduction
 Examples of biotechnological products
 Protein and peptides
 Monoclonal antibodies
 Oligonucleotides
 Vaccines (immunotherapy)
 Gene therapies
 Reference
2

3. What is Pharmacokinetics study (PK)…?
It is a branch of Pharmacology which deals with the study of
Absorption, Distribution, Metabolism, Excreation/Elimination.
Pharmacokinetics is a study of “What the body does to the drug”
What is Pharmacodynamics study (PD)...?
In Greek Pharmcon – Drug
Dynamics – Action
Pharmacodynamics is the study of biochemical and physiologic effect
of drug.
Pharmacodynamics is a study of “What the drug does to the body”
3

4. Biotechnology Drugs :
 Pharmaceutical biotechnology consist of the combination of
two branch which are “Pharmaceutical science” and
“Biotechnology”
Pharmaceutical science : It can be simply define as the branch
of science that deals with the formulation compounding and
dispensing of drugs.
Biotechnology : Biotechnology drug differ from Pharmaceutical
drugs in that they use biotechnology as a means for
manufacturing, which involves the manipulation of
microorganism, such as bacteria, or biological substance, like
enzymes, to perform a specific process.
Ex- Antibiotics, vaccines etc.
4

5. Biotechnological products
 Biotechnology can be defined as application of technology using
the living organisms to obtain useful products.
 The products made by the biotechnology process include,
pharmaceuticals (medicine), food, and water purification, genetic
known as biotechnological products.
Types of biotechnology products :-
 Industrial and Environmental biotechnology
 Medical / pharmaceutical biotechnology
 Agricultural biotechnology
 Diagnostic research biotechnology
5

6. 6

7. 7

8. Examples of biotechnological
products
1. Proteins and peptides
2. Monoclonal antibodies
3. Oligonucleotides
4. Vaccines (immunotherapy)
5. Gene therapies
8

9. 1. Protein and peptides
 Protein : Protein are the large organic compound made of amino acids
arranged in linear chain and joined together by peptide bonds.
Protein > 50 amino acids
Molecular weight above 5000
 Peptide : These are short polymer formed from the linking in a
defined order of amino acids
Peptide < 50 amino acids
Molecular weight less than 5000
9

10. • Protein are the most aboundant macromolecules in the living cells,
occurring in all cells and all parts of cell.
• Scientific advances in molecular and cell biology have resulted in the
development of two new biotechnologies.
• The first utilizes RECOMBINANT DNA to produce protein product.
• The second technology is HYBRIDOMA TECHNOLOGY. Various protein and
peptide drug are epidermal growth factor, tissue plasminogen activator
10

11.  The size and complexity of protein or peptide drugs require extensive design
and engineering of manufacturing and control processes to produce the drug in
large quantities with consistent quality.
 The 3D structure of a protein or peptide drugs is important for its
pharmacodynamics activity, such as 1º amino acid, 2º (alpha helix), 3º (special
relationship of 2º structure), 4º orientation of subunits must be considered.
 Small changes in manufacturing process may affects the sequence of the
resulting protein, but are more likely affects to the structure, yield, or activity
of protein or drug products.
11

12. Application of peptide
12

13. 2. Monoclonal antibodies
 Antibody or immunoglobulin's are protein molecules produced by a specialized
group of cells called B-lymphocytes in mammals.
 An antibody is a protein produced by white blood cells and used by the immune
system to identify and neutralize foreign objects like bacteria, viruses and foreign
substances. Each antibody recognizes a specific antigen unique to its target.
Monoclonal antibodies (mAb) are antibodies that are identical because they were
produced by one type of immune cell, all clones of a single parent cell.
Polyclonal antibodies are antibodies that are derived from different cell lines.
13

14.  An antigen can be a foreign molecule that interacts with the
cells of the immune system, triggering an immune response.
 The molecules on the antigens to which the antibodies attach
themselves are called epitopes.
 The region of the antibody which binds to the epitope is
called a paratope.
14

15.  The power of mAb lies in their highly specific binding of only
one antigenic determinant. As a result, mAb drugs, targeting
agents, and diagnostic are creating new ways to treat and
diagnose.
 Monoclonal antibodies can also target and deliver
toxin specifically to cancer cells and destroy them
while sparing normal cells and important detectors
used in laboratory diagnostics.
15

16. Application of monoclonal
antibodies
Cancer treatment
Pregnancy test
Diagnosis of HIV infection
16

17. 3. Oligonucleotides
 Antisense drugs consist of nucleotides linked together in short DNA or
RNA sequence known as oligonucleotides.
 Antisense oligonucleotides drugs, are drugs that seek to block DNA
transcription or RNA translation in order to moderate many disease
processes.
 Oligonucleotides are chemically synthesized by using phosphoramidite.
 The oligonucleotide chain proceeds in the direction of 3’ to 5’ terminus.
17

18.  Antisense oligonucleotides are the molecules made of synthetic genetic
material, which interact with the natural genetic material that codes the
information for production of proteins.
 Antisense RNA prevents protein translation of certain mRNA strands by
binding to them.
 Antisense DNA can used to target a specific complementary RNA.
Examples
1. Mipomersen for high cholesterol.
2. Affinitak and a Genasense against cancer.
3. AV 1-6002 and AV 1-6003 for the treatment of Hemorrhagic fever.
18

19. Applications of oligonucleotides
Antisense
oligonucle
o-tide
therapy
CVS
Therapeuti
cs
CNS
Therapeuti
cs
Oncolog
y
Inflammatio
n
Therapeutics
As
Antiviral
and
antibacter
ial agents
19

20. 4. Vaccines (Immunotherapy)
 A vaccine is a biological preparations that improves immunity to a particular
disease.
 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.
 Vaccines are dead or inactivated organisms or purified product derived from
them. The different types of vaccines are ;
a) Traditional vaccines
b) Innovative vaccines
20

21. a) Traditional vaccines
1. Killed
2. Live, attenuated
3. Toxoid
4. Subunit
21

22. a) Traditional vaccines
1. Killed :– Some vaccines contain killed, but previously virulent,
microorganism that have been destroyed with chemicals, heat, radioactivity or
antibiotics.
Examples : are influenza, cholera, polio, hepatitis A, and rabies.
2. Live, attenuated :- Some vaccines contain live, attenuated
microorganisms. Many of these are active viruses that have been cultivated under
conditions that disable their virulent properties or that use closely related but less
dangerous organisms to produce a broad immune response.
Example : are yellow fever, measles, mumps.
22

23. 3. Toxoid - Toxoid vaccines are made from inactivated toxic
compound that cause illness rather than the microorganism.
Examples : are Tetanus and Diphtheria.
4. Subunit – Protein subunit – Rather than introducing an
inactivated or attenuated microorganism to an immune system (which
would constitute a whole agent vaccine), a fragment of it can create an
immune response.
Example : Meningococcal disease and Pneumococcal disease.
23

24. b) Innovative vaccines
1. Conjugate vaccines
2. Recombinant vector vaccine
3. T-cell receptor peptide vaccine
4. Valence
5. Heterotypic
24

25. b) Innovative vaccines
1. Conjugate vaccines - Certain bacteria have polysaccharide outer coats
that are poorly immunogenic. By linking these outer coats to protein(e.g. toxin),
the immune system can be led to recognize the polysaccharide as if it were a
protein antigen.
Example : Hib (Haemophilus influenza type b) disease
2. Recombinant vector vaccine - By combining the physiology of one
microorganism and the DNA of the other, immunity can be created against
diseases that have complex infection process.
Example : Hepatitis B
25

26. 3. T-cell receptor peptide vaccine - They show the modulation of cytokine
production and improve cell mediated immunity and are under development.
Use : to stimulate antitumor T cell
4. Valence - a) Monovalent- Use to immunize against single antigen.
b) Multivalent- Used to immunize against two or more
microorganism.
Uses : Hepatitis A, Hepatitis B, mumps, rubella, diphtheria, chickenpox etc
5. Heterotypic - Vaccines that are pathogens of other animals that either do not
cause disease or cause mild disease in the organism being treated.
Use : diphtheria 26

27. 5. Gene therapy
 A technique for correcting defective genes that are responsible for
disease development.
 Gene Therapy in this use of DNA as a pharmaceutical agent to treat
disease.
 It drives its name from that DNA can be used to supplement or alter
genes within an individual cells as a therapy to treat disease.
 The most common form of gene therapy involves using DNA that
encoded a functional, therapeutic gene to replace a mutated gene.
27

28. Approaches for gene therapy
1.Gene modification
a. Replacement therapy
b. Corrective gene therapy
2.Gene transfer
a. Physical (Microinjection, Gene gun, naked DNA, Electroporation)
b. Chemical (Liposomes, Cationic liposomes, Oligonucleotides etc.)
c. Biological (Viral vector , mammalian artificial chromosomes)
3. Gene transfer in specific cell lines
a. Somatic gene therapy
b. Germ line gene therapy
4.Eugenic approach(gene insertion) 28

29. 1. Gene modification
 In replacement therapy , a defective gene is inserted somewhere in the genome so that its
product could replace that of a defective gene.
 Corrective gene therapy, requires replacement of a mutant or a part of it with a normal
sequence.
 This can be achieved by using recombinant technology.
 Another form of corrective therapy involves the suppression of a particular mutation by a
transfer RNA that is introduced into the cell.
2. Gene transfer
 Gene transfer can be used for improvement of a specific disease. For example, introducing a
growth hormone gene to increase the height.
 This gene transfer into cell can be brought about by physical, chemical and biological
methods. 29

30. 3. Gene transfer to specific cell lines
 Somatic gene therapy, which has emerged as a new approach for the treatment
of a variety of genetic and a acquired diseases, involves the insertion of genes
into specific somatic cells.
 Germ line therapy, injection or insertion of genes into cells into germ cells into
fertilized eggs is known germ line therapy which
differs from somatic cell therapy.
4. Eugenic approach( gene insertion)
Eugenic is a movement that is aimed at improving the genetic composition of
human race.
Eugenic approach is brought about by inserting to alter or improve complex
traits of a person.
30

31. Reference
 Leon Shargel, Susanna WU- Pong, “Applied biopharmaceutics and pharmacokinetics”,
Sixth edition 2012, Cataloging-in-publication, page no. 505-513.
 S. P. Vyas and V. K. Dixit, “Pharmaceutical Biotechnology”, CBS Publication, page no.
402-409.
 http://en.m.wikipedia.org
 http://www.youtube.com
 http://www.google.com
31

32. 32

33. 33