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A FOOD & Drug Administration advisory body recently conducted a public meeting on its evaluation of data concerning the safety of a new human blood-thinner drug that would become the first derived from genetically engineered animals to be approved for use in the U.S.
Developed by the Massachusetts-based biotechnology company GTC Biotherapeutics, it involves the production of a human antithrombin drug -- ATryn -- in the milk of genetically engineered goats. The drug acts as a natural blood thinner in those with a hereditary disorder that affects about one in every 5,000 people and make them vulnerable to blood clots.
The product is to be administered intravenously for a limited time in such patients as they undergo surgery or give birth.
To make the drug, GTC scientists inserted DNA for the human antithrombin drug into single-cell embryos of dairy goats. The goat embryos then were inserted into the wombs of surrogate does. The first of the goats were termed "founders," and their offspring also carry the gene. The females produce high levels of antithrombin in their milk, from which the protein is collected and purified.
GTC's production of the drug is derived from a herd of approximately 200 dairy goats in central Massachusetts.
Until now, antithrombin has been produced from blood products collected from human donors, and scientists hope production can be done more safely and in larger volumes by using genetically engineered goats.
ATryn was approved by the European Commission in 2006 and is already being marketed in Europe. | 
