Angela Zimm Bloomberg News
Published: Monday, December 4, 2000
What do you get when you cross a goat with DNA science on a New England dairy farm?
At Genzyme Transgenics Corp. in rural Massachusetts, you get an animal that makes milk for medicines.
Genzyme Transgenics has been genetically engineering goats to produce milk that contains antibodies and other proteins to treat diseases ranging from AIDS to arthritis. More than a genomics-age novelty, these goats are being developed as low-cost way of making today's biologic medicines, said Sandra Nusinoff Lehrman, the company's chief executive.
"Goats have the ability to make a lot of protein with relatively modest capital investment," Lehrman said. Goats are easy to manage, reproduce quickly and the start-up cost of making drugs from goat's milk can be less than 5 percent of the investment needed to build a factory, the company said.
Today Genzyme Transgenics' goats produce 12 experimental medicines for nine companies. The company, a subsidiary of Cambridge, Massachusetts-based Genzyme Corp., expects to seek U.S. Food and Drug Administration approval next year of its first goat-produced drug, antithrombin III, a naturally occurring human protein that acts as an anticoagulant.
Some analysts say the company could be well positioned to take advantage of a boom in drug development as the mapping of the human genome leads to the discovery of more gene-based proteins and demand for new ways to make them.
"I think the technology is quite promising," said Elise Wang, an analyst for investment banker UBS Warburg. "It provides an alternative method of producing proteins at high yields. A lot of companies are looking for alternatives."
Genzyme Transgenics turns goats into medicine factories using a process called "transgenics," taking DNA from the genes of one species and implanting it into the genes of another.
To make a goat that produces a particular human protein in its milk, the gene for that protein is combined with a milk- producing gene to create a "transgene." The transgene is then injected into a fertilized goat embryo that is then implanted in a female goat.
The result is transgenic offspring, the females of which produce the desired protein in their milk. Transgenic offspring are then mated to build a herd of the same protein-producing goats, since they can transmit the gene for the protein from generation to generation. About one in four of the female offspring is born with the ability to produce the desired protein in her milk.
Analysts say Genzyme Transgenics is farther along with transgenic animal technology than rivals including PPL Therapeutics Plc, the U.K. company that cloned Dolly the sheep, and Pharming NV, a Netherlands-based company that develops transgenic cows.
Biotechnology companies now make many of their antibodies and proteins in cell cultures grown in large incubators called bioreactors. To build a typical bioreactor plant producing 400 to 500 kilograms of a protein per year costs about $350 million, said Greg Liposky, vice president of operations at Genzyme Transgenics' 380-acre farm and dairy. About 40 percent, or $140 million, represents the cost of just the bioreactor.
Genzyme Transgenics can create the same capacity for $10 million, with one barn, 250 goats, an attached milking facility and a processing center where the protein is extracted from the milk, Liposky said.
The company is also applying its transgenic technology in other animals, including the development of a malaria vaccine in the milk of mice.
Genzyme Transgenics has yet to turn a profit. It generated $69 million in revenue last year, of which $13.2 million came from partnerships with drug companies.
Some of those collaborations include developing goat's-milk versions of Johnson & Johnson's rheumatoid arthritis drug Remicade, Elan Pharmaceuticals Inc.'s experimental multiple sclerosis treatment Antegren, an experimental AIDS treatment for Progenics Pharmaceuticals, and several experimental antibodies in partnerships with Bristol-Myers Squibb Co., BASF AG/Knoll Pharmaceuticals, Abgenix Inc. and Alexion Pharmaceuticals Inc.
The FDA hasn't indicated how it will handle review of goat's-milk versions
of drugs, such as Remicade, that it has previously approved based on conventional