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DRUGS / ANIMALS: Down on the biopharm

------------------------------- GENET-news -------------------------------
TITLE:  Down on the biopharm
SOURCE: LabTechnologist, France
AUTHOR: Anna Lewcock
DATE:   17.05.2007

Down on the biopharm

17/05/2007 - The critical role transgenic animals could play in the
future of biopharmaceutical production has been tackled by a task force
in a new report out this week.

The potential such animals could have in the manufacture of
biopharmaceutical proteins has been one of the major incentives driving
investigation and creation of transgenic animals, and with therapeutic
products derived from these genetically modified creatures now beginning
to gain regulatory approval, commercial interest is in the technique is
hotting up.

The significant cost savings to be had through using transgenic
livestock instead of traditional methods of protein production have been
well documented, and comparisons make for stark reading.

To illustrate, experts have estimated that producing a single gram of
therapeutic protein using traditional cell lines such as Chinese Hamster
Ovary (CHO) cells can cost anywhere from $300 to $3,000 (?221 to ?2210).

In contrast, using a transgenic goat to produce the protein in milk
drops the cost to $20-$105 per gram, and transgenic hen eggs are even
cheaper, working out at around $0.1-$0.25 per gram of protein

The initial capital expenditure is also somewhat less intensive using
transgenic livestock, with the cost of constructing a new facility based
on traditional cell-based techniques hitting $150m - $400m, compared
with the cost of a transgenic goat or cow at $10,000 - $50,000, or a
transgenic chicken coming in at $1,000.

"With moderate alterations in production practices, it is possible to
take advantage of the tremendous protein-producing capabilities of
domestic livestock," states the report, published this week by the
Council for Agricultural Science and Technology (CAST).

"Biopharming, the production of biopharmaceuticals using domestic
livestock, can have significant advantages compared with other
production methods in terms of safety, biological activity, and
production costs."

Back in June 2006 the European Medicines Agency (EMEA) gave the very
first European approval of a transgenically produced protein product.
ATryn, produced by US firm GTC Biotherapeutics, is produced in the milk
of goats that have a transgene for human antithrombin, and is used as an
anticoagulant to treat a rare congenital disease.

This first step to move trangenically produced protein products out onto
the market could prove to be the just the tip of the ice-berg, with all
eyes on ATryn to see how it fares out in the cold reality of the marketplace.

According to the chairperson of the CAST task force, Carol Keefer, wide-
scale commercial adoption of protein production using transgenic animals
is getting closer and closer, with GTC's product cutting a path for
other biotech companies.

"I think as soon as ATryn goes to market and proves acceptable and
profitable, more pharma companies will explore the use of transgenic
animals for protein production," she told

Already there are many companies investing in research into producing
bioproducts through transgenic livestock. The majority are still in
research stages, but US company Avigenics and Netherlands-based firm
Pharming currently both have products in clinical trials.

Aside from the economic cost benefits biopharming appears to promise,
the procedure can also offer other distinct advantages over current cell-
based production methods.

For example, using transgenic animals to produce biopharmaceuticals
currently harvested from human tissues represents a safer technique in
terms of preventing transmission of human diseases such as HIV/AIDS or
Creuzfeldt-Jakob disease, say the report authors.

In some cases, using transgenic animals can also lead to production of a
better protein, said Keefer, i.e. a protein more similar to the version
naturally produced in humans.

"Proteins are modified during production in the cell, and transgenic
animals can do these modifications in a manner more similar to the human-
produced protein than other production systems such as yeast or bacteria."

Despite the apparent attractions of biopharming, the report acknowledges
that it is critical to establish economic feasibility of the process
before it will be adopted by drug firms. For example, issues surrounding
protein purification can seriously affect the ultimate economics and
commercialisation of a final product.

Feasibility concerns or lack of funding have caused some commercial
pharma projects applying these new technologies to be scrapped or
delayed on a purely economic basis.

"These are business issues caused not necessarily by technical
challenges but by unknown factors that arise as new technologies develop
without an established track record or sufficient guidelines for
completing the necessary regulatory steps," states the report.

"In fact, regulatory guidelines are being developed concurrently with
the establishment of the new technology, creating uncertainty within the
business community as to the costs and timelines associated with
recombinant protein production."

While transgenic livestock are likely to play an increasingly
significant role in the production of therapeutic proteins, Keefer was
by no means suggesting that it would to do away with other production
methods altogether.

"Each production method has its advantages and disadvantages," she said.

"Depending on the protein, the amount of protein needed (based on market
demand and dosage required), and the activity of the protein, a company
would choose the best suited system. [For example] if the therapeutic
protein could have an effect on the physiology of the transgenic animal,
then you would either modify the protein so it was inactive during
production, or perhaps choose another system."

Despite this, the authors of the report clearly have very high
expectations of biopharming and the part it will play in the production
of new medications to treat human diseases. The unique possibilities
that transgenic livestock present in this area are too big a prospect to
be ignored, and the authors call for "continued support of research by
both government and commercial entities...such that additional promising
biotherapies can be developed."

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