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Biopharmaceuticals,background & introductionbackground & introduction
Gary WalshGary Walsh
University of LimerickUniversity of Limerick
Cork, Jan. 20Cork, Jan. 20thth, 2009, 2009
Points for discussion
I.I. Some basicsSome basics
2
II.II. The biopharmaceutical sectorThe biopharmaceutical sector
I. Some Basics
�� What are biopharmaceuticals?What are biopharmaceuticals?
�� Protein structureProtein structure
3
Protein structureProtein structure
�� Genetic engineeringGenetic engineering
�� Biopharmaceutical manufacture: an Biopharmaceutical manufacture: an
introductory overviewintroductory overview
What are Biopharmaceuticals?
Modern biotech. medicines:Modern biotech. medicines:
�� Proteins produced by genetic engineeringProteins produced by genetic engineering
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�� Monoclonal antibodyMonoclonal antibody--based productsbased products
�� Nucleic acidNucleic acid--based productsbased products
�� Engineered cell/tissueEngineered cell/tissue--based productsbased products
Protein structure
�� Linear chains of amino acidsLinear chains of amino acids
�� 20 naturally occuring amino acids20 naturally occuring amino acids
�� Amino acid sequence dictated by geneAmino acid sequence dictated by gene
�� Assume a specific 3Assume a specific 3--D conformationD conformation
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n
PTM
(e.g. glycosylation)
Genetic Engineering
Human gene
(e.g. insulin)
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(e.g. insulin)
Producer cell
(e.g. E. coli)
Human protein
Why produce proteins via genetic
engineering?
�� Produce protein in large amountsProduce protein in large amounts
Produce ‘safe’ productProduce ‘safe’ product
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�� Produce ‘safe’ productProduce ‘safe’ product
�� Produce engineered productsProduce engineered products
Expression systems
Expression Expression
E. coliE. coli
Animal Animal
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Expression Expression
SystemsSystems
TransgenicsTransgenics
S. cerevisiaeS. cerevisiaeAnimal Animal
cell linescell lines
Biopharm production, an overview
Producer cell line/
Expression system
Upstream processing
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Upstream processing
Downstream processing
Formulation
Traditional products v
biopharmaceuticals
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II. The biopharmaceutical Sector
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II. The biopharmaceutical Sector
Biotech companies, global overview
12
Nat. Biotech. (2007), 7, p706
Globally, sector driven by US companiesGlobally, sector driven by US companies
Biopharmaceuticals: Global Status
�� 27 years since ‘Humulin’27 years since ‘Humulin’
�� 200 approved for human use200 approved for human use
�� All except 2 are proteinsAll except 2 are proteins
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�� All except 2 are proteinsAll except 2 are proteins
�� Global market value ~ $40 billionGlobal market value ~ $40 billion
�� More than 2,000 in clinical trialsMore than 2,000 in clinical trials
Approved proteins
– hormones
– monoclonal antibodies (Mabs)
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– interferons/ interleukins
– blood-related products
– vaccines
The future
15
Nat. Biotech. (2006) 12, p 1466
Points discussed
�� Some basics Some basics
The biopharmaceutical sectorThe biopharmaceutical sector
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�� The biopharmaceutical sectorThe biopharmaceutical sector
Thank you!
