Pharmaceutical Biotech 1-9-10

8/8/2019 Pharmaceutical Biotech 1-9-10

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Overview

IntroductionManufacturing processes of Biotech compounds

used in Pharmaceutical and Biopharmaceutical

fieldsPharmaceutical Development Strategies

Gene therapy

Formulation for a pharmaceutical and

b iopharmaceutical



C hapter I: INTR ODUC TION



W hat is Pharmaceutical?

Pharmaceutical substances form the backbone of modernmedicinal therapy

Most traditional pharmaceuticals are low molecular weightorganic chemicals (Table 1.1).

A range of pharmaceutical substances (e.g. hormones andblood products) are produced by/extracted from biological

sources.



Ta b le 1.1 S ome traditional pharmaceutical substances that are generallyproduced by direct chemical synthesis

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W hat is Biopharmaceutical?

µBiopharmaceutical¶ and µProducts of pharmaceuticalbiotechnology¶ or µBiotechnology medicines¶

µBiologic¶ refers to any pharmaceutical product produced bybiotechnological endeavour. In pharmaceutical circles,µbiologic¶ generally refers to medicinal products derived fromblood, as well as vaccines, toxins and allergen products.

µBiotechnology¶ has a much broader and long-establishedmeaning. Essentially, it refers to the use of biological systems(e.g. cells or tissues) or biological molecules (e.g. enzymes orantibodies) for/in the manufacture of commercial products.



Pharmaceutical BiotechnologyModern biotechnology have had major impact on Pharmaceutical and

Biopharmaceutical fields.C ultivation of cells used in pharmaceutical and b iopharmaceutical

fields.

Gene Disruption

Expression of Genes

Mutasynthesis

Recom b inant DN A technology

Hyb ridoma technology

X-crystallization application

Formulation for a pharmaceutical and b iopharmaceutical

Gene therapy



R educing cost

Accelerating the Drug Development Process: development andmanufacture of therapeutic proteins and nucleotides.

Maintaining High Standards in Quality Assurance

W hat is the Impact of Pharmaceutical Biotechnology on theDrug Development Process?



Biomedical research continues to broaden our understanding of themolecular mechanisms underlining both health and disease.

R esearch undertaken since the 1950s has pinpointed a host of proteinsproduced naturally in the body that have obvious therapeuticapplications.

Example

Interferons and interleukins (which regulate the immune response)Growth factors, such as erythropoietin (EPO; which stimulates

red blood cell production)

The age of b iopharmaceuticals



The pharmaceutical potential of regulatory molecules was generallyappreciated, their widespread medical application was in most casesrendered impractical due to the tiny quantities in which they werenaturally produced.

The advent of recombinant DNA technology (genetic engineering)and monoclonal antibody technology (hybridoma technology)overcame many such difficulties, and marked the beginning of a newera of the pharmaceutical sciences.




R ecombinant DNA technology has had a fourfold positive impactupon the production of pharmaceutically important proteins:

It overcomes the problem of source availability.It overcomes problems of product safety.

It provides an alternative to direct extraction frominappropriate/dangerous source material.It facilitates the generation of engineered therapeutic proteinsdisplaying some clinical advantage over the native proteinproduct.




Biopharmaceuticals: current status and future prospects

By mid 2006, some 160 b iopharmaceutical products hadgained marketing approval in the US A and/or EU (Ta b le 1.3)

Protein- b ased therapeutic agents: hormones, b loodfactors and throm b olytic agents, as well as vaccines andmonoclonal anti b odies.

Nucleic-acid- b ased products: µVitravene¶, an antisenseoligonucleotide, and µMacugen¶, an aptamer.



Many of the initial biopharmaceuticals approved were simplereplacement proteins (e.g. blood factors and human insulin).

The vast majority of approved recombinant proteins have

been produced in the bacterium E. coli

, the yeast S. cerevisiae

orin animal cell lines (most notably Chinese hamster ovary (CHO)cells or baby hamster kidney (BHK) cells.




Although most biopharmaceuticals approved to date are intended forhuman use, a number of products destined for veterinary applicationhave also come on the market.

R ecombinant bovine GH (Somatotrophin), which was approved in the

USA in the early 1990s and used to increase milk yields from dairycattle.

Veterinary biopharmaceuticals include a range of recombinant vaccinesand an interferon-based product.




Ta b le 1.3 Summary categorization of b iopharmaceuticals approved for general medical use in the EU and/or US A b y 2006



W hat can you do by the end of this course?

Overview the development of pharmaceutical biotechnology

Outline the applications of biotechnology in pharmaceutical

and biopharmaceutical fields

Understand how to drug design and drug development

Understand the R egulations in pharmaceutical manufacturing



W hat topics will be covered?

Pharmaceutical and Biopharmaceutical productionPharmaceutical practiceM anufacturing processes of Biotech compounds used in Pharmaceuticaland Biopharmaceutical fieldsPharmaceutical Development Strategies

Nucleic-acid and cell-based therapeutics



REVISI ONS



Procaryotic and Eucaryotic Cells in Biotech Production

Fermentation of microorganisms or the use of yeast and plants in theproduction of important pharmaceuticals has been well established.

The promises of genomics in drug discovery and drug production,which were eagerly embraced in the mid-1990s, have now been fulfilledin many areas.

A systematic integration of technologies results in a superior output of data and information, and thereby enhances our understanding of biological function ± drug discovery and development is hence facing a

new age.



Bacterial strains, especially Actinomycetes have been used in biotechproduction and drug discovery for years.

The productivity of yeast and other fungi in a variety of different processeshas improved significantly since genetic methods have been introduced.

Transgenic plant Biotech

Procaryotic and Eucaryotic Cells in Biotech Production



Figure 1 . Convergence of genomic and clinical knowledge in postgenomic new drug development and healthcare



The overall discovery/development process can be divided intofour convenient steps

Target Selection and Validation

Definition of Target Product Profile

Drug Discovery and Development

Choice Between Small-molecule and Macromolecule Platforms

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Pharmaceutical Biotech 1-9-10

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