Pharma-Bioengineering

Prof. Dr. Oliver Kayser

Technical University Dortmund

Technical Biochemistry

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16.03.2015 Pharm. Biotechnologie (MSc) 2

Definition

Biotechnology is the integration of natural

sciences and engineering sciences in order to

achieve the application of organisms, cells,

parts thereof and molecular analogues for

products and services.

How does Biotechnology work?

technology

Chemical -

Physical -

Nano -

Production -

Material -

Material -

Information -

Communication -

application

Pharma/ Medicine Food Chemistry Enviroment Farming Industrial

product / process

Diagnostics Drugs Vaccines Enzymes Flavours Pollution Sustainable processes New crops Functional food Biochips Displays

Biotechnology - Gene technology

Biotechnology

Using natural sources and

biotransformation

Using technical principals for

manufacturing biotech products

Working with genetically non-

manipulated cells

„Classic genetics“ allowed !

No manipulation of isolated DNA

Keywords:

Up- and Downstream,

Fermentation,

Bioprocessing, Bioreaktor

Genetechnology

Isolation and manipulation of genes

Production of „recombinant“ DNA

Introduction of new, heterologic

DNA and functions

Cloning, Recombination,

Transgenesis, Genetherapy

DNA

(genome)

enzyme

(protein) Molecules

educts Molecules

products

Principles of bioprocessing

What is Pharma Biotechnology?

Pharma-Biotechnology covers all sciences and

technologies that are involved in discovery,

process of development, characterisation,

production and regulation of pharmaceutical

biotech products

(Suggestion of EAPB e.V.)

www.eapb.org

How does the drug development process works?

Phase 1 Phase 2 Phase 3 Phase 4 P reclinical Phase

Biotechnology

Pharmaceutical Sciences

Analytics, Chemistry,

Stability, Protein Formulation

Toxicology, Pharmacology

Pharmacokinetic,

Drug Regulation Protein Formulation

Biopharmacy

Documentation

Bioprocessing

Scaling Up

GMP facilities

FDA/EMEA-Guidelines for

Production

Economic aspects

Pre-Clinical IND Phase 1

Phase 2

Phase 3

Market Approval

Launch

From Lead to Launch:

• 7 - 12 Years

• €200M to > €400M

Probability of Success 1/10 - 1/5

1/3

1/2

9/10

95%

Earnings Potential: €1M / day

Some recombinant proteins approved for human use ($60 billion-2006)

Protein Company Disorder

Factor VIII Baxter, Bayer Hemophilia A

Factor IX Genetics Institute Hemophilia B

Tissue plasminogen activator (TPA)

Genetech Acute myocardial infarction

Insulin Eli Lilly, Novo Nordisk Diabetes mellitus

Human growth hormone

Eli Lilly, Genetech, Upjohn, Novo Nordisk

GH deficiency in children (dwarfism)

Erythropoietin Amgen, Ortho Biotech Anemia

DNase I Genetech Cystic fibrosis

Various interferons (IFN)

Schering, Biogen, Chiron,

Genetech

Hepatitis B and C, multiple sclerosis

Some therapeutic monoclonal antibodies approved for human use

Type of antibody Company Therapeutic use

Mouse, Humanized Ortho Biotech, Protein Design, Hoffmann-LaRoche

Prevents kidney transplant rejection

Chimeric Centocor Prevents blood clots

Chimeric Genetech, Hoffmann-LaRoche

Non-Hodgkin lymphoma

Humanized (Herceptin)

Genetech HER2-positive breast cancers

Humanized Am Home Prod, Celltech, Schering, Millen. Pharm.

Certain leukemias

Humanized Genetech Asthma

Interdisciplinary field Pharma-Biotechnology Genetics

• Gene technology

• Gene sequecning

Microbiology/ Immunology

• Bacteriology

• Mycology Pharmaceutical Biology

• Natural Product Chemistry

• System Biology

Pharmaceutical Technology

• Drug Targeting

• Drug Delivery

• Aseptic production

Biochemistry

• Enzymology

• Molecular biology

Pharmaceutical Chemistry

• Peptide chemistry

• Analytics

• Drug design

• Protein chemistry Chemistry

• Organic Chemistry

• Physical Chemistry

Biochemical engineering

• Downstream processing

• Upstream Processing

Bioprocessing

• Process Design

• Unit Operations

Synthetic Biology

• Gene design

• Pathway architecture

Biology

• Cytology

• Physiology

• Cell culturing

Pharmaceutical

Biotechnology

General considerations I

Disadvantages

• Production under aseptic conditions

• Complex media

• Instabile cellular systems

• problems in waste management

• Strict safety guidelines in production

• Problematic downstreaming

General considerations II

Advantages

• Using sustainable sources

• Reducing energy consumption by experimental

conditions at atmosphere pressure and normal

temperatures

• Minimizing of pollution

• Reduction of synthetic steps in classic organic

chemistry

• Reduction of unwanted metabolites

• One-step (bio)synthesis in manufacturing process

Expansion of Pharma Companies

Ernst & Young, Biotech Report 2013

Investments in Pharmaceutical Biotechnology

US Biotechnology: Commercial leaders and other companies, 2011-2013

EU Biotechnology: Commercial leaders and other companies, 2011-2013

FDA Approvals, 1996-2012

Turn over with genetically produced recombinant drugs

Pipeline by indication Pipeline by year

Poduction Systems used, 1980-2010

Ferrer-Miralles et al. 2010 Microbial Cell Factories 8:17, 1-8

More...

BIO http//www.bio.org

Bio.Com http://www.bio.com

EAPB http://www.eapb.org

EFB http://www.dechema.de/efb

Ernst&Young http://www.ernst-young.de oder http://www.ey.com

IFPMA http://www.pharmweb.net

IMS-Health http://www.imshealth.com

ISB http://www.i-s-b.org/

Verband forschender Arzneimittelhersteller

http://www.vfa.de

http://www.ey.com/Publication/vwLUAssets/B

eyond_borders/$FILE/Beyond_borders.pdf