prof. dr. oliver kayser technical university dortmund ... bio-engineering/pharma...prof. dr. oliver...
TRANSCRIPT
Pharma-Bioengineering
Prof. Dr. Oliver Kayser
Technical University Dortmund
Technical Biochemistry
Recommended Literature G
en
tech
nik
– B
iote
chn
ik, K
ap. I
.1
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