GTC Biotherapeutics (fka GTCB)

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For old times sake…
Biotech Company Cracks Gene Process

http://www.michigansmalltech.com/news/articleprint.asp?strContentid=4664CC04-8160-49F6-A239-3EE1086A...

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Mar 21, 2005
By Tom Henderson

ANN ARBOR -- GeneWorks Inc., an Ann Arbor agricultural biotechnology firm, says it has made dramatic breakthroughs recently in efforts to grow human genes in white leghorn chicken eggs, a process called transgenics that promises to greatly reduce the cost of drug discovery and the treatment of people with defective genes.

William MacArthur, founder and president of GeneWorks, announced in February that the company had confirmed the presence of a gene encoding its third pharmaceutical protein in the semen of two genetically modified roosters. The presence of the gene in the semen means the roosters will be able to breed the gene in future generations of chicks.

The company previously identified 11 roosters carrying a gene that encoded the company's first proprietary protein, and nine carrying a second gene encoding another protein. One protein helps grow blood cells in people undergoing chemotherapy, one reduces cholesterol and one helps treat those who have suffered from strokes or heart attacks.

The roosters are descended from embryos that were injected with snippets of HIV engineered to contain human genes. MacArthur says the HIV it is using has been made harmless "by removing 90 percent of its genomes."

GeneWorks hopes to breed sufficient flocks of the gene-bearing chicks within a year to be able to sell 100-liter batches of purified egg whites that contain the protein to drugmakers. For competitive purposes, he declined to disclose the protein. MacArthur said the company should begin generating profits once it also begins licensing its technology to big pharmaceutical firms.

"Getting the roosters to breed true is like tapping an oil well," he said. "We'll really be able to ramp up production, now. We're past the proof of concept."

The breakthroughs come at a crucial time for the company. It has spent much of the $18 million it has raised in venture capital. In the late 1990s, Sloan Ventures of Birmingham helped it raise $3 million. A second wave of financing that totaled $15 million came in 2000 from a Manhattan investment firm.

GeneWorks spent $4 million on what Dr. Jerry Dodgson, a professor at Michigan State and the former department head of microbiology and molecular genetics, says is a state-of-the-art chicken-breeding facility and lab in Bridgewater Township southwest of the company's headquarters in Ann Arbor.

The facility includes 40,000 square feet of breeding and lab space in two buildings in the middle of 156 acres. It has the capacity to raise 14,000 chickens, but currently houses 900 adult hens, 600 adult roosters and hundreds of chicks.

With money running out, GeneWorks downsized its headquarters and in November reduced its work force from 23 to 8.

MacArthur said the company is currently seeking more funding. While biotech was hot for venture capitalists in the go-go 1990s, funding has largely dried up since.

"Venture capital is really tough in biotech, now, as you know," says MacArthur.

According to Dodgson, using transgenic chickens to produce pharmaceutical proteins is far cheaper than the current industry practice of using Chinese hamster ovary cells in huge arrays of cell-culture vats that can cost hundreds of millions of dollars to install.

"The goal for a long time has been to efficiently express proteins in egg whites," Dodgson said. "Eggs have been used for vaccines for years, so they're the logical source for other biological projects. And you can grow thousands or millions of chickens in a cost-effective manner."

Dodgson said that while GeneWorks' chicken-breeding facility might give it a leg up on its competitors, it still faces stiff challenges from AviGenics, Inc., a company based in Athens, Ga., and by a consortium headed by the Roslin Institute in Scotland, famous for its cloned sheep, Dolly.

"The struggle will be to express a sufficient amount of a useful protein to make it economically attractive," he says.
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