Transgenics is a process whereby DNA is taken from one species and implanted into the genetic structure of another. Genzyme Transgenics, which became GTC Biothereapeutics (GTCB) in 2002, uses this process to develop and produce human therapeutics in the milk of animals. After GTCB successfully cloned their first goat, testing showed that the animal was, indeed, transgenic, and the goat's milk tested positive for antithrombin III -the human protein. ATIII is a protein found in human plasma involved in helping to regulate blood clotting.
A transgenic form of antithrombin III, a/k/a recombinant human antithrombin III [rhATIII], is produced "by inserting human DNA into the cells of goats so that the targeted protein is excreted in the milk of the female offspring."
(One of GTC Biotherapeutics’ transgenic goats (T&G File Photo/DAN GOULD))
A little history:
Genzyme Transgenics Corporation was originally a subsidiary of Genzyme Corporation (GENZ), and ultimately morphed into GTC Biotherapeutics in June 2002. And, simply put, it is no longer tied to Genzyme Corporation. GTCB is an entirely separate entity.
The idea for an animal-milk protein was the brainchild of researchers at Biogen (BIIB) in Cambridge, Mass. (more than 22 plus years ago), and one of their scientist's, Harry Meade, pushed the idea that the udder of a farm animal "could be programmed to produce useful molecules too complicated and delicate to be manufactured in the large fermenting tanks used in most biotech plants. And it could generate the valuable substances in far larger quantities."
Meade left Biogen and was hired by Genzyme Corporation, whereby this technology spun its way into Genzyme Transgenics Corporation.
GTC Biotherapeutics has conducted clinical trials of rhATIII in coagulation disorders, but in 2006, Europe's top drug-regulation committee rejected the company's anti-clotting drug, ATryn®, on the basis that the drug had been tested in too few people to determine if it was safe and effective. ATryn® was eventually approved for use in the European Union for hereditary antithrombin deficiency (HD) patients undergoing surgical procedures. LEO Pharma A/S is currently commercializing and developing ATryn® in Europe.
Back in the USA, GTCB announced in June 2008 that it had signed a collaboration deal with Ovation Pharmaceuticals, Inc., to develop and market ATryn® in the United States. The agreement includes $257 million in potential payments to GTCB for meeting clinical, regulatory, and sales milestones.
GTCB is also milking other applications of its goat-based rhATIII technology, although the projected market initially for ATryn® is small- perhaps an estimated $50 million.
In September 2008, the FDA assigned priority review to GTCB's BLA for ATryn®. On January 9, 2009, the company announced that an FDA advisory panel (the Blood Products Advisory Committee of the U.S. Food and Drug Administration) gave "thumbs up" to the drug for the prevention and treatment of venous thromboembolism in hereditary antithrombin deficient patients undergoing surgery or childbirth procedures, which, if approved by the FDA, would be an historical event and tipping point in the progression of bio-pharma technology. The FDA usually renders its decisions based upon their advisory panels' recommendations. A final decision on ATryn® is due by February 7, 2009.
Looks like, what with pro-stem cell heavyweight proponent President-Elect Barack Obama about to take Office, that genetically-based biotech's time has finally come. The FDA has also just issued Final Guidance on Regulating Genetically Engineered Animals, "“Genetic engineering is a cutting edge technology that holds substantial promise for improving the health and well being of people...", says Randall Lutter, Ph.D., FDA deputy commissioner for policy.
However, this is not the only exciting thing for GTCB. In fact, it's "small potatoes" compared to what is now becoming a highly conceivable reality (assuming an affirmative FDA final decision) in regard to a main objective of the company.
You see, antithrombin currently costs $1.72 per international unit, or more than $100,000 per pound, when traditionally produced by purifying antithrombin from human blood. And, GTCB's herd of transgenic goats, 1,500 of them* (located on a highly-secure 19th-century "pharm" in Charlton, Mass -near Framingham), each produce more than 1.5 pounds of protein per year: or, more than $150,000 worth per goat- per year! This translates, potentially, to a top-end stream (no pun intended) of over $200 million per year in revenue for the herd- and, easily makes GTC Therapeutics' goats (transgenic or otherwise), the world's most valuable. And, of course, the company can easily breed more goats, at any point, to meet demand.
[* see bottom of page 7 in 10k filing]
My only question is: what do they eat?
"Udderly" amazing stuff. And GTC Biotherapeutics, after a very long and strange journey, is finally poised to get its multi-million dollar herd of goats moving off that farm in Charlton, and onto the rich and plentifully fertile plains of big pharma.
"Get along, Yip. Yip. Get along! Whoa-there, little fella, get back in the pack! Now there ya go..."
GT McDuffy
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