(Protein-based medicines) Pharmaceuticals (Chemically-based drugs)

mutebabiesΒιοτεχνολογία

6 Δεκ 2012 (πριν από 4 χρόνια και 10 μήνες)

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Biotechnology…A Promise Come True

Max Saráchaga

Medical Director

Amgen

2

2

Treating Disease

Treating People,

3

People are part of an astonishing variety of life.

We

re all made of the same material.

Typical Cell

Protein

Sugar

Fat

Chronic Kidney Disease

People with kidney disease do not make
enough of the EPO protein, produced by the
kidneys to stimulate production of red blood
cells.

Erythropoietin (EPO) Protein

When complex processes are all working precisely, we are healthy…

But for many people, something goes wrong. Disease is the result.

Different Treatments





______
________


____________
________



Different Causes of Disease,

Biotech Medicines:

Live,

& Complex

Large

Biologics Differ From Traditional Drugs

in Both Complexity and Sensitivity

Manufacturing

Unique bank of living cells;

Unlikely to achieve identical copy

Predictable chemical & reagent reaction;

Identical copy can be made

Characterization

Many products well
-
characterized;

Correlation of structure
-
function elusive

Easy to fully characterize

Stability

Sensitive to storage and

handling conditions

More stable

Immunogenicity

Higher potential

Lower potential

Structure

Complex

Simple

Stability

Complex Degradation

Stable

Variability

Heterogeneous ‘product’

Single defined structure

Biologics

(Protein
-
based medicines)

Pharmaceuticals

(Chemically
-
based drugs)

Size

Large

Small

Properties

Typical Protein Production Process

Typical Protein Production Process

Typical Protein Production Process

Typical Protein Production Process

Fermentation units.
Host cells

thrive in these vats (also called

bioreactors
) filled with a life
-

sustaining bath of nutrients.

Fermentation

is the
science of growing host
cells in large quantities, so
that the gene inside them
produces the protein that
we want in large quantities.

Typical Protein Production Process

Put gene into

DNA vector

Identify and isolate
gene

that will produce a
medically useful protein

Transfer into Host Cell

Expression

Screening/Selection

DNA vectors
are used
to move genes into new
organisms. They are
generally plasmids
(pieces of bacterial DNA
that can live and
replicate outside the
chromosome).

Chinese hamster ovary (CHO) cells

were introduced in the early 1960s as
an ideal cell line for genetic studies.

Genes won

t produce
proteins outside of a living
cell, so we have to insert
our gene into a living cell (a

host cell

).

Biotech Medicine:
The final protein
must have precisely the right amino acid
sequence, folded into precisely the right
three
-
dimensional shape.

Downstream

Extraction/

Purification/

Dilution

We must validate that
downstream processing
continues to remove viruses,
DNA and toxins from the
protein product stream.

Validation testing

may be defined as

the act of proving that any procedure,
process, equipment, material, activity or
system leads to the expected results.


Biocomparables
:

Similar, not Identical to Original Products

Different cell lines

Different manufacturing processes

....but not identical

Biocomparables

are similar...

Impact of small differences in either biological or manufacturing process
could lead to different clinical efficacy and safety for patients

No therapeutic substitution

Immunogenicity differences

10

We must always keep patients in mind.



Amgen: A Biotechnology
Pioneer


Founded in 1980, Amgen
was one of the first
biotechnology companies to
successfully discover,
develop, and make protein
-
based medicines


Today, we’re leaders in the
next wave of innovation:


Developing therapies in
multiple modalities


Driving cutting
-
edge
research and development


Continuing to advance the
science of biotechnological
manufacturing

Our Worldwide Presence and a Leader In
Biotechnology Manufacturing

The Netherlands

Belgium

Ireland

Spain

Switzerland

Juncos, Puerto Rico

Washington, DC

Louisville, KY

West Greenwich, RI

Cambridge, MA

Toronto, ON

Thousand Oaks, CA

Seattle, WA

Bothell, WA

Hungary

United
Kingdom

Denmark

Portugal

Italy

Germany

Norway

France

Czech Republic

Sweden

Austria

Poland

Australia

Boulder, CO

Longmont, CO

Finland

South San Francisco, CA

Slovenia

Slovakia

Lithuania

Latvia

Estonia

Luxembourg

Burnaby, BC

United Arab Emirates

Mexico City, Mexico

Hong Kong

India

Russia

Greece

Brazil

Japan

Bulgaria

Romania

Turkey

Woburn, MA


54

Countries by 2011


75 Countries by 2015

35% of worldwide manufacturing capacity

Our Products

Oncology, Nephrology, Bone, Metabolism, Inflammation


For additional information about Amgen products, including
important safety information, please visit www.amgen.com

Research and Development at
Amgen

Guiding Principles


Focus on serious illness


Be modality independent


Assess efficacy in patients


Seamless integration from
research through
commercialization

Pioneering Science

Delivers Vital Medicines

For more information, visit www.amgen.com

Our Mission : To serve Patients