Pharmaceutical Biotechnology in Drug Development

By Muhammad Sajid Hamid Akash, Kanwal Rehman, Kanwal Irshad and Shuqing Chen

Pharmaceutical Biotechnology in Drug Development summarizes key concepts and the latest developments of biotechnology applied to the development of biopharmaceuticals. Chapters present a comprehensive collection of introductory biotechnology technologies and their modern concepts and cover pharmacokinetic and pharmacodynamic behavior of biopharmaceuticals and modification techniques of amino acids and nucleic acid. Other sections focus on topics such as gene therapy, immunological preparations and nanoparticles which are the major contributions of pharmaceutical biotechnology. Final chapters discuss emerging techniques in the field of pharmaceutical biotechnology to meet current patient and health care demand.

This book is an essential reference useful for pharmaceutical scientists, clinicians and academic researchers who want easy access to up-to-date practices of pharmaceutical biotechnology. Corporate researchers will also benefit from this book’s succinct and objective content structure.

• Includes key concepts at the foundation of the technology and relevant for protein therapeutics
• Explains how advances in other areas such as genomics, proteomics and high-throughput screening have paved the way for exploring new avenues of drug discovery
• Covers the importance of biotechnology in the development of new biopharmaceuticals, along with their pharmacodynamics and pharmacokinetics

• Language: English
• Publisher: Academic Press
• Release date: Apr 21, 2023
• ISBN: 9780443191367

Muhammad Sajid Hamid Akash

Dr. Akash is currently working as associate professor and chairman at Department of Pharmaceutical Chemistry, Government College University Faisalabad (GCUF), Pakistan. He got his bachelor and master’s degrees in pharmacy from Bahauddin Zakariya University Multan, Pakistan and PhD degree in Pharmaceutical Analysis from Zhejiang University, China. Currently, he is focusing on investigating the effect of EDCs on various molecular and metabolic pathways that are involved in metabolic disorders and the treatment strategies of these EDCs-induced metabolic disorders funded by Higher Education Commission (HEC) of Pakistan. His Research work is the cutting edge and worthy of extension in developing countries like Pakistan, where it can be most beneficial and cost-effective especially for diabetic patients. Based on his significant scientific contributions in the field of Pharmaceutical Sciences, he has been awarded “Research Productivity Award” from Pakistan Council for Science and Technology (PCST), PAS Gold Medal from Pakistan Academy of Sciences and A.R. Shakoori Gold Medal from Zoological Society of Pakistan.

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Chapter 1: Modern concepts of pharmaceutical biotechnology in drug development

Abstract

Pharmaceutical biotechnology is a branch of biomedical sciences that uses novel technologies for production, formulation, and synthesis of biological substances from the living organisms, which act as drug molecules for the treatment and/or prevention of various diseases and/or syndromes. Pharmaceutical biotechnology has a major role in drug discovery and development for the understanding of many diseases and their novel treatment strategies and therapeutic interventions. In this chapter, we have described in detail the modern concepts of different branches of biotechnology in relation to their role in drug discovery and development. Moreover, we have also discussed the different techniques, including gel electrophoresis, western blotting, polymerase chain reaction, molecular cloning, tissue culture, microarrays, deoxyribonucleic acid sequencing, and enzyme-linked immunosorbent assay that are frequently used during the process of drug development. We have also comprehensively described the new emerging drug classes such as monoclonal antibodies, small drug molecules, protein drugs, therapeutic hormones and enzymes, cytokines, and antisense drugs that are biotechnologically derived from the genetically modified organisms.

Keywords

Biological substances; Biotech products; Biotechnological techniques; Polymerase chain reaction

Introduction

Biotechnology can be defined as the use of living organisms or biological processes for the development and production of products that are beneficial for the improvement of humans' health. This term can be defined as the utilization of living organisms to improve the human health and status. In the modern era of biotechnology, it is defined as the production of hybrid genes by shifting the desired gene from one organism to another organism that usually not possess that particular gene expression. In the past years, biotechnology has been used to produce different species of plants by cross-pollination, which combat with different diseases and has good quality. It also plays an important role in the production of animals by crossbreeding, which possess better traits than their parents. Moreover, it also plays a significant role for the production of various products such as beer, yogurt, cheese, bread, and wine by the process of fermentation using microorganisms [1].

The term biologics is referred to as the pharmaceutical products that are produced by using different biotechnological processes and techniques. In the field of pharmacy, this term can be defined as the medicinal product produced by using biotechnological techniques. Moreover, it can be demonstrated as the medicinal substances derived from the blood, toxins, allergen products, and vaccines. The term biopharmaceutical was used in 1980s and introduced a unique class of biotechnologically derived therapeutic proteins, which is generated by using novel biotechnological techniques such as recombinant deoxyribonucleic acid (DNA) technology and monoclonal antibodies (mAbs). A large number of biopharmaceutical products including therapeutic proteins and peptides are produced with the help of genetic engineering. This term also covers the nucleic acid–based substances such as DNA and ribonucleic acid (RNA)–based substances and the biological products based on the whole cell [2].

The scope of biotechnology covers all the aspects of agriculture and health-related issues. Nowadays, biotechnology has been used to improve the expression of different targeted genes and to discover the medicinal products. Moreover, biotechnology helps to understand the genetic composition and their sequence in human beings to identify and treat the hereditary diseases. Multiple innovative techniques are used in the diagnosis, prevention, and treatment of diseases, including DNA sequencing, cell culture, interference RNA, biopharmaceuticals, genome editing, gene sequencing, tissue engineering, stem cells for regenerative medicines, and antibiotics. Medical biotechnology to develop novel medicinal products has become a basic foundation in medical science [3].

Different biotechnological techniques are based on the novelty of different branches of the science, including cell biology, molecular biology, and biochemistry. This field imparts the beneficial impacts on different era of life such as medical technology, food processing, treatment of waste, and agriculture. There are two sides in biotechnology: one is to modify the DNA and the other is the transfer gene from one organism to another organism.

There are several other ways to define the term biotechnology, which are as follows:

• Biotechnology can be described as the use of biological agents such as cellular components and microorganisms for the welfare of mankind [4].

• Biotechnology consists of two terms, bio and technology. The term bio deals with the life, and the term technology deals with the applications of various techniques on living organisms for the production of biotechnologically derived products.

• It can be referred as the commercial employment of various living organisms such as plants, animals, and microorganisms using various technological tools to develop commercial substances such as antibiotics, vaccines, and vitamins that are helpful to improve the health of human.

• The Universities Press Dictionary of Biology (UPDB) has defined the term biotechnology as the applications of biotechnological techniques for agricultural, industrial, and medical purposes [5].

• Biotechnology can also be defined as the use of living organisms such as fungi, algae, yeast, bacteria, and cells of animals and plants, isolated components of living matter within the system, and biological processes for the production of biotechnologically derived commercial products [6].

Branches of biotechnology

Biotechnology is an emerged and/or multidisciplinary filed of the science, and the activities involved in it have significant effects on human wellness, such as the production of nutrition-enriched food, protection of biospheres from harms, and improving the health quality. However, the areas in which biotechnology has main roles include human health, agriculture, fisheries, forestry, environment, horticulture, animal husbandry, parental dispute, food processing and mining, etc. There are different branches of biotechnology, which have been discussed in the proceeding sections.

Animal biotechnology

This is a branch of biotechnology that deals with the development of transgenic animals to increase the quality and quantity of milk and meat production. These products biotechnologically derived from transgenic animals are also resistant to various kind of diseases. Animal biotechnology exhibits the in vitro fertilization and transfer of embryos into different animals as well as in humans [7]. It is pertinent that biotechnology is very important to understand the treatment of many genetic disorders. Proteomics, bioinformatics, and genomics are used in animal biotechnology. Transgenic techniques are used to improve the quality and quantity of milk and meat production. Transgenic techniques are also used to develop several knockout experimental animal models to understand the occurrence and pathogenesis of several diseases and genetic disorders. Animal biotechnology is also used to produce specific proteins, which possess significant therapeutic potential against several diseases and disorders. For the cloning of transgenic animals, nuclear transfer technology is preferred, while for the cloning of farm animals, genetic engineering is usually performed. This technique is also used for the treatment and management of various zoonotic diseases such as classical swine fever, foot-and-mouth diseases, bovine spongiform encephalopathy, and avian flu [8,9]. There are many biotechnologically derived therapeutic products, including vaccines especially DNA or genetically engineered vaccines, which are available in the market to treat various diseases and genetic disorders.

Currently, animal biotechnology has focused on the treatment of pet animals with gene therapy because many biotechnologies in human clinical trials have priorly used the transgenic animals during the process of drug development and preclinical trials. This is because of the reason that several human diseases have similar pathophysiology as that in animals, particularly cats, rats, mice, or dogs [8,9]. Moreover, there are many diagnostic techniques, including fluorescent in situ hybridization, polymerase chain reaction (PCR) and its modifications, enzyme-linked immunosorbent assays (ELISAs), biosensors, and newer biochip-based technologies that are used for the diagnostic purpose in veterinary and medical sciences [8,9].

Medical biotechnology

This branch of biotechnology is involved in the development of different technologies, which are used in medical and pharmaceutical industries. In medical biotechnology, living cells and the cellular material are used for the research and investigational purposes, and to develop the diagnostic and biotechnologically derived pharmaceutical and/or therapeutic agents for the prevention and treatment of different diseases. Moreover, this branch of biotechnology has also involved in the development of marketed insect-resistant crops, market microbial pesticides, and environmental clean-up techniques [3,10]. Furthermore, it deals with the production of large amount of drug substances, blood serum proteins, antibodies, hormones, including insulin and interferon, and vaccines for various diseases, including chicken pox, measles, rabies, polio, etc.

Industrial biotechnology

Industrial biotechnology has focused on the wide applications of industrial processes, including bioprocessing to produce the bio-based products such as plastics, chemicals, and fuels from the feedstocks which is renewable. Moreover, at industrial level, the processes and technologies based on the microbes, enzymes, microorganisms, and genetic engineering are used for the development of many varieties of commercial products. This branch deals with the production of very useful organic materials such as acetic acid, acetone, chloroform, glycerin, citric acid, etc., and antibiotics such as penicillin, mitomycin, streptomycin, etc., by using different microorganisms such as bacteria and fungi. Now, there is a need to introduce novel industrial manufacturing procedures that should be better, economic, pollution-free, and environmentally friendly. Nowadays, several molecular biological techniques are used to enhance the efficiency and reduce the harmful effects of many industrial processes such as chemical manufacturing, textile, and production of paper and pulp.

Environmental biotechnology

Environmental biotechnology deals with the removal of toxins from waste and industrial effluents. Moreover, it has involved in the prevention of plant diseases and in the treatment of sewage water by using different biological agents such as bacteria, virus, fungi, etc. The aim of environmental biotechnology is the use of optimal resources and biomass recycling, energy recovery, and reducing the waste production by using modern processes and technologies. Moreover, to optimize the exploitation of biotechnological techniques, there must be emphasis on the waste management and treatment, soil conservation, bioremediation of water and land, rehabilitation of land, reforestation, and afforestation. The applications of environmental biotechnology play important role in the protection of environmental integrity for long-term ecological security. This field has a vital role in the development of new solutions for the prevention, reservation, and mitigation of environmental damage with the help of living organisms. A complex population of naturally existing microorganisms helps in the treatment of sewage. All living organisms ingest nutrients to live and produce waste material as a by-product. Different organisms require different nutrients, such as some bacteria obtain their nutrients from chemical components of waste products. Therefore, they are used in the treatment of waste material. With the increase of environmental pollution, there is a need to develop analytical devices that can detect the hazardous compounds in land, air, and water. Recombinant DNA technology and bioremediation have provided possibilities to reduce environmental pollution.

Plant biotechnology

Plant biotechnology deals with the combination of two techniques, including genetic engineering and tissue culture. In this branch, we deal with the development of high-quality transgenic plants that are resistant to harsh environmental conditions, biotic and abiotic stresses, rescue of embryo, development of haploid cells, clonal multiplication, etc. In plant biotechnology, a set of techniques are used to modify the plants for specific opportunities or needs, such as a single crop may provide nutrition, sustainable food, new opportunities for jobs, and environmental protection. But it is a very difficult and complex challenge to develop a suitable plant. Plant biotechnology assists in the development of novel traits and varieties, including genetics and genomes, marker-assisted selection, and genetic engineered crops.

The scientists have developed a large yield of plants with low cost, which are highly resistant to diseases and pests by using biotechnological techniques. Now the researchers have focused on the development of plants that combat with environmental stresses such as drought, and also able to utilize the soil nutrients more efficiently. Moreover, scientists are trying to develop certain types of plants, which have adequate nutritional value and capacity to produce pharmaceuticals by using different biotechnological tools.

Color classification of branches of biotechnology

The classification of biotechnology branches is based on colors like a rainbow in which each color represents a specific subsector.

Blue biotechnology

Blue biotechnology deals with marine and waterborne organisms and has a wide range of applications. This branch of biotechnology is used for the protection of marine organisms from harmful diseases within the water [11]. Many aquatic animals such as shellfish, fish, and sponges are used for the production of different products. However, nowadays blue biotechnology has been using an incredible variety of the living organisms from lakes, earth oceans, streams, and rivers. The scientist has extracted useful substances from the decades of sponges that possess therapeutic potential against AIDS. Moreover, cod liver oil has been used as a supplement. It is a very modern and unique field because of its adaptive nature [12].

Bioinformatics

The combination of computer and biotechnology is called bioinformatics. It deals with findings and critical analysis of the given data, which are related with biotechnology. It is used for the development of many drugs, crops fertility, and to produce resistance in plants to combat with different diseases. It has a significant role in different areas of science such as functional and structural genomics and proteomics, which are considered to be a major contributor in the field of biotechnology and pharmaceutical sector [11]. There are many applications of bioinformatics, such as genome sequencing, gene identification, determination of gene function, determination of protein structures, development and designing of drugs, phylogeny, organism's identification, vaccine designing, and understanding of genome complexity. With the help of bioinformatics, time-consuming projects can be done rapidly such as mapping of human genome [13].

Green biotechnology

This term is widely used in agriculture field for the development of new varieties of plants and for the production of biofertilizers and biopesticides. The micropropagation is used in this branch for the production of good quality plants and crops. It deals with the development of transgenic plants that can grow under specific environmental conditions in the presence of certain chemicals [11]. It has wide applications in agriculture for the production of high-quality food and to enhance the nutritional benefits and yield. It also secures the global ecological health and life. The basic principle of this branch lies on the manufacturing of products, which can diminish the pollution in environment. In this field, modified crops are produced such as the production of rice crops with induced resistance against bacterial, light, increased level of β-carotene and tolerance of submergence.

Red biotechnology

Red biotechnology is also known as biopharma or medical biotechnology, which deals with the production of large variety of novel drugs, vaccines, antibiotics, and development of regenerative therapies, molecular diagnostic technique, and genetic engineering techniques for the treatment of diseases by the genetic manipulation. It also deals with stem cell therapy for the growth of organs and to cure damaged and diseased tissues in the organisms [11]. By the utilization of biotechnological techniques, many biopharmaceuticals are produced including proteins, nucleic acids, DNA, RNA, and antibodies. These products are used for diagnostic and in vivo therapeutic purposes. These products are different from other pharmaceutical products because they are developed by biotechnology methods rather than direct extraction. The first biopharmaceutical product is insulin, which is produced by the recombinant DNA biotechnology and used as medicine.

White biotechnology

It is also known as industrial biotechnology, which deals with industrial processes. This helps in the production of plastics, vehicle parts and fuel, fiber in clothing industry, and production of new chemicals [11]. This branch of biotechnology uses the living organisms such as molds, plants, yeast, and bacteria for the synthesis of degradable products. It requires less energy and produces less waste products. It is used for the production of metabolites, waste treatments, synthesis of biocontrol agents, and bio-based energy and fuels. The activities of white biotechnology are involved with industrial microbiology for the isolation of microorganism from the nature, improvements in the yield of products, screening for the product formulation, cultures' maintenance, and recovery of services and products.

Gold biotechnology

Gold biotechnology is referred as computational biology, which deals with biological problems via computational techniques for rapid organization and analysis of biological data. It also refers to the study of nanobiotechnological science. The biotechnology and nanotechnology have been emerged to form nanobiotechnology, which has commercial and biomedical applications in various areas of science such as catalysis, biomedicine, electronics, and agriculture because of unique physical, chemical, magnetic, and electronic properties [14]. Nanobiotechnology also provides a chance to introduce new substances and methodology for better understanding of interactions of nanomaterials with intracellular processes, structure, and environment [15].

Yellow biotechnology

It is related to the production of food such as beer, wine, cheese, and yogurt by the fermentation [16]. It can be defined as the utilization of biotechnology-based approaches for the exploitation of insects or models derived from insects, cells, organs, molecules, and development of beneficial products for human or provide services for agriculture, and medical and industrial applications. Because of molecular and taxonomic diversity of insects, they are known as indispensable biological resources that are now used as a source of valuable products. However, pest insects compete with human for food while vector insects transmit several diseases such as malaria and plague that threatened the humanity and killed millions of people throughout the history. It utilizes the biotechnological tools for the development of sustainable method to control the pest, insects, and vectors [17].

Gray biotechnology

Gray biotechnology deals with the environment for the maintenance of biodiversity and removal of many kinds of pollutants such as heavy metals and hydrocarbons from the environment by using microorganisms and some plants [16]. This field has concerned with the protection of flora and fauna, control of pollution, and management of waste by the utilization of microorganisms and bioremedial technology for the removal of pollutants. This includes the interactions between the microorganisms and environment, and the effects of microorganisms on the water, soil, and air pollution [18].

Violet biotechnology

It deals with the philosophical and ethical issues, and laws that address with biotechnology [16]. This branch of biotechnology is devoted to regulate and resolve the issues. This branch also provides a platform for biotechnology-related problems. The aim of all other branches of biotechnologies is to increase the number of inventions. This branch also deals with issues related to patent invention, which is the most hectic