Anti-clotting drug from genetically engineered goats

Washington, United States, January 8: One of the first of its kind, anti-clotting drug, named ATryn, developed from genetically engineered goats is on its way to approval by the government. It has already been given the stamp of safety by the Food and Drug Administration.

The latest medication has been made in order to make life easier for people, who suffer from a rare hereditary disease that makes them susceptible to blood clots.

GTC Biotherapeutics, a Massachusetts biotechnology company, has developed ATryn. The drug is manufactured by manipulating the genesdefine of goats. This is done in such a way that the goats produce milk, which is high in antithrombin, a protein that makes human blood thinner naturally.

The drug has been made by scientists at the GTC by inserting the human antithrombin protein into single cell embryos of goats. These embryos were then put into the wombs of surrogate mothers who produced goats that possessed the new characteristics. The protein is gathered from the milk of the goat and then purified.

But deficiency of this protein has been seen in almost one in 5,000 people. This makes them vulnerable to painful clots that can break and reach the lungs or brain through the bloodstream. This can cause dangerous consequences.

If pregnant women suffer from such a problem, it is very likely that due to formation of blood clots in the placentadefine, there is a miscarriage or stillbirth.

Currently, patients with antithrombin deficiency are administered conventional blood thinners. This mode of treatment would still continue as ATryn will only be administered to patients, who are undergoing surgery or having a delivery. This is because the risk of clot formation is higher at these times.

The drug will be given to patients through intravenous infusion for a specific time period before and after the procedures.

The pros and cons of ATryn will be seen into by the scientific advisors at the FDA. They will then make a further recommendation for approval of the drug.

FDA spokeswoman Siobhan DeLancey said, “It's the first time we've held an advisory committee meeting on any product from a genetically engineered animal.”

If the drug is approved, then this would be a significant leap in the area of making medicines by altering genes of living organisms.

“The real dramatic thing that is happening here is that we've been able to reduce some very clever science to the practical level of producing a drug that's safe and efficacious,” said Geoffrey Cox, Chairman GTC.