ATryn
Encyclopedia
ATryn is the brand name of the anticoagulant
antithrombin
manufactured by the Massachusetts
-based U.S. company GTC Biotherapeutics. It is made from the milk of goat
s that have been genetically modified
to produce human antithrombin, a plasma protein
with anticoagulant properties. Microinjection
was used to insert human antithrombin genes into the cell nucleus
of their embryo
s. ATryn is the first medicine produced using genetically engineered animals. GTC states that one genetically modified goat can produce the same amount of antithrombin in a year as 90,000 blood donations. GTC chose goats for the process because they reproduce more rapidly than cattle
and produce more protein than rabbits or mice.
On February 6, 2009, ATryn was approved by the U.S. Food and Drug Administration (FDA) for treatment of patients with hereditary antithrombin deficiency
who are undergoing surgical or childbirth procedures. Along with the approval from the FDA's pharmaceutical regulatory board, the Center for Veterinary Medicine
of the FDA also approved the genetic makeup of the goats that are used to manufacture ATryn. GTC has the sole rights to sell ATryn in the United States, and the drug is available in the U.S. market. Earlier in 2006, the European Medicines Agency
(EMEA) initially rejected and, after an appeal from GTC, approved the drug for use in the European Union
countries.
According to Tom Newberry, the spokesperson for GTC, the company plans to acquire additional approval for treatment of those with non-hereditary antithrombin deficiency.
The Humane Society of the United States
has said of the process used to manufacture ATryn, "It is a mechanistic use of animals that seems to perpetuate the notion of their being merely tools for human use rather than sentient creatures." However, the genetic change has no known ill-effects on the host animal.
Anticoagulant
An anticoagulant is a substance that prevents coagulation of blood. A group of pharmaceuticals called anticoagulants can be used in vivo as a medication for thrombotic disorders. Some anticoagulants are used in medical equipment, such as test tubes, blood transfusion bags, and renal dialysis...
antithrombin
Antithrombin
Antithrombin is a small protein molecule that inactivates several enzymes of the coagulation system. Antithrombin is a glycoprotein produced by the liver and consists of 432 amino acids. It contains three disulfide bonds and a total of four possible glycosylation sites...
manufactured by the Massachusetts
Massachusetts
The Commonwealth of Massachusetts is a state in the New England region of the northeastern United States of America. It is bordered by Rhode Island and Connecticut to the south, New York to the west, and Vermont and New Hampshire to the north; at its east lies the Atlantic Ocean. As of the 2010...
-based U.S. company GTC Biotherapeutics. It is made from the milk of goat
Goat
The domestic goat is a subspecies of goat domesticated from the wild goat of southwest Asia and Eastern Europe. The goat is a member of the Bovidae family and is closely related to the sheep as both are in the goat-antelope subfamily Caprinae. There are over three hundred distinct breeds of...
s that have been genetically modified
Genetic engineering
Genetic engineering, also called genetic modification, is the direct human manipulation of an organism's genome using modern DNA technology. It involves the introduction of foreign DNA or synthetic genes into the organism of interest...
to produce human antithrombin, a plasma protein
Blood proteins
Blood proteins, also termed serum proteins or plasma proteins, are proteins found in blood plasma. Serum total protein in blood is 7g/dl...
with anticoagulant properties. Microinjection
Microinjection
Microinjection refers to the process of using a glass micropipette to insert substances at a microscopic or borderline macroscopic level into a single living cell. It is a simple mechanical process in which a needle roughly 0.5 to 5 micrometers in diameter penetrates the cell membrane and/or the...
was used to insert human antithrombin genes into the cell nucleus
Cell nucleus
In cell biology, the nucleus is a membrane-enclosed organelle found in eukaryotic cells. It contains most of the cell's genetic material, organized as multiple long linear DNA molecules in complex with a large variety of proteins, such as histones, to form chromosomes. The genes within these...
of their embryo
Embryo
An embryo is a multicellular diploid eukaryote in its earliest stage of development, from the time of first cell division until birth, hatching, or germination...
s. ATryn is the first medicine produced using genetically engineered animals. GTC states that one genetically modified goat can produce the same amount of antithrombin in a year as 90,000 blood donations. GTC chose goats for the process because they reproduce more rapidly than cattle
Cattle
Cattle are the most common type of large domesticated ungulates. They are a prominent modern member of the subfamily Bovinae, are the most widespread species of the genus Bos, and are most commonly classified collectively as Bos primigenius...
and produce more protein than rabbits or mice.
On February 6, 2009, ATryn was approved by the U.S. Food and Drug Administration (FDA) for treatment of patients with hereditary antithrombin deficiency
Antithrombin III deficiency
Antithrombin III deficiency is a rare hereditary disorder that generally comes to light when a patient suffers recurrent venous thrombosis and pulmonary embolism...
who are undergoing surgical or childbirth procedures. Along with the approval from the FDA's pharmaceutical regulatory board, the Center for Veterinary Medicine
Center for Veterinary Medicine
The Center for Veterinary Medicine is a branch of the U.S. Food and Drug Administration that regulates the manufacture and distribution of food, food additives, and drugs that will be given to animals. These include animals from which human foods are derived, as well as food additives and drugs...
of the FDA also approved the genetic makeup of the goats that are used to manufacture ATryn. GTC has the sole rights to sell ATryn in the United States, and the drug is available in the U.S. market. Earlier in 2006, the European Medicines Agency
European Medicines Agency
The European Medicines Agency is a European agency for the evaluation of medicinal products. From 1995 to 2004, the European Medicines Agency was known as European Agency for the Evaluation of Medicinal Products.Roughly parallel to the U.S...
(EMEA) initially rejected and, after an appeal from GTC, approved the drug for use in the European Union
European Union
The European Union is an economic and political union of 27 independent member states which are located primarily in Europe. The EU traces its origins from the European Coal and Steel Community and the European Economic Community , formed by six countries in 1958...
countries.
According to Tom Newberry, the spokesperson for GTC, the company plans to acquire additional approval for treatment of those with non-hereditary antithrombin deficiency.
The Humane Society of the United States
Humane Society of the United States
The Humane Society of the United States , based in Washington, D.C., is the largest animal advocacy organization in the world. In 2009, HSUS reported assets of over US$160 million....
has said of the process used to manufacture ATryn, "It is a mechanistic use of animals that seems to perpetuate the notion of their being merely tools for human use rather than sentient creatures." However, the genetic change has no known ill-effects on the host animal.