1022-7954/02/3801- $27.00 © 2002
Russian Journal of Genetics, Vol. 38, No. 1, 2002, pp. 1–14. Translated from Genetika, Vol. 38, No. 1, 2002, pp. 5–21.
Original Russian Text Copyright © 2002 by Goldman, Kadulin, Razin.
The pharmaceutical industry is an intensely devel-
oping ﬁeld because of the increasing demand for high-
quality pharmaceuticals and diagnostic preparations.
The progress in this ﬁeld is based on successful devel-
opment of effective drugs and use of innovative techno-
logical approaches in their production. One of these
approaches is preparation of biologically active human
proteins (hormones, enzymes, immunomodulators,
antibodies, biostimulators, clotting and growth factors)
from milk of transgenic animals [1–4].
These proteins were traditionally isolated from
donor blood. Therefore due to blood deﬁcit and low
concentrations of the proteins in blood, they were
obtained in small amounts, which did not fulﬁll even a
small proportion of the market demand for the pharma-
ceuticals. In addition, drug preparation from donor
blood is potentially hazardous because of virus contam-
ination (hepatitis, AIDS).
Microorganisms cannot be used for production of
most biologically active high-quality human proteins in
sufﬁcient amounts and of required quality because they
are unable to implement posttranslational modiﬁca-
tions of complex eukaryotic proteins. Many human
proteins produced in microbial systems are insoluble,
which hinders their isolation. In addition, the prepara-
tions obtained by microbial synthesis are known to be
allergic if they contain even trace amounts of bacterial
Production of medicinal substances by the animal
cells in bioreactors is extremely expensive. The cost of
a modern industrial bioreactor is over $100 million.
Bioreactors devised to obtain particular types of pro-
teins are poorly adaptable for other purposes. There is
also the problem of genome instability in cultivated
cells. All this makes, the biologically active human pro-
teins expensive pharmaceuticals (Table 1).
The far more promising technology of the 21st cen-
tury is human protein production by transgenic ani-
New-generation medicinal substances isolated from
transgenic-animal milk, a product natural for humans,
are biologically safe. This is a highly economical and
organic mode of the manufacture, which is not power-
consuming and does not require special facilities
because the transgenic animals are maintained in ordi-
nary agricultural farms .
Transgenic Goats in the World Pharmaceutical Industry
of the 21st Century
I. L. Goldman
, S. G. Kadulin
, and S. V. Razin
Institute of Gene Biology, Russian Academy of Science, Moscow, 117334 Russia;
fax: (095) 135-41-05; e-mail: firstname.lastname@example.org
University of Oslo, Center for Medical Science, Moscow, 117334 Russia
Received March 11, 2001
—In many developed countries, isolation of human pharmaceutical proteins from milk of genetically
modiﬁed animals is currently a priority. One of the ﬁrst commercial pharmaceuticals obtained from the milk of
transgenic goats, an anticoagulant antithrombin III, developed by Genzyme Transgenic Corporation, an Amer-
ican biotechnological company, will appear on the pharmaceutical market in the nearest future. In this review,
we discuss the role of fundamental science in the development of this ﬁeld of the pharmaceutical industry.
Estimation of the annual world demand and the cost of some pharmaceutical human proteins that are supposed to
be obtained from transgenic animalbioreactors* 
FVIII FIX protein C ATIII fibrinogen HSA
Amount 304 g 4 kg 10 kg 21 kg 150 kg 315000 kg
Actual cost for 1 g (US $) 2900000 40000 10000 7000 1000 3.56
Market value (US $ million) 882 160 100 150 150 1120
* Information of the United States Red Cross.