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Start-Ups at Cutting Edge of Science Innovations

By 1993, says Brum, Vertex had identified a likely anti-AIDS drug candidate--code-named VX-478--and signed an $42 million agreement with Glaxo-Wellcome to market and develop the compound under the brand name Amprenavir. Clinical trials began in February, 1995, and approval from the Food and Drug Administration (FDA) is expected by the end of 1998.

Automation and Information

Three blocks away from Vertex, at 640 Memorial Drive, another new pharmaceutical company is developing tools and techniques that are redefining the way the industry searches for drugs.

Millennium Pharmaceuticals, which is less than five years old but already has, three other locations in Cambridge, began by focusing on genomics, the science of understanding not only the sequence of all of the genes in human chromosomes, but also the function of the protein each gene codes for and under what circumstances each gene is expressed.

Such Knowledge is critical for pharmaceutical companies, which must constantly seek out new drugs to interact with new targets to find new cures, and Millennium already has lucrative alliances with six members of "big pharma," including Monsanto and Eli Lilly.

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"What we hope to achieve is working with the pharmaceuticals industry to provide them with novel targets, gene products, which we feel are ripe for the development of a novel therapeutic, whether it be a small molecule or a protein therapeutic," says Robert Tepper, chief scientific officer for pharmaceuticals at Millennium.

But like Vertex, Millennium itself aspires to become a full-fledged pharmaceutical company and has several proprietary drug discovery efforts underway.

"I think we've anticipated the impact that the efforts going on in the Human Genome Project will have on biomedical science, and we have built tools...to come to bear with dealing with this enormous amount of information," Tepper says.

"These technologies include very state-of-the-art information systems, which really are relatively new to biomedical research and came of age as part of sequencing the human genome," he says, noting that computational techniques and databases as large as the human genome are commonplace in the financial industry.

Tepper says he believes bioinformatics, as these technologies are collectively called, are paving the way for the future of biotechnology.

"While foreign to most biologists and perhaps chemists, and, in fact, not taught in normal biomedical education, these become the most important tools for the next wave of biomedical research," he says.

In addition to information systems, Millennium has been an avid proponent of automation, such as the techniques of combinatorial chemistry employed by Vertex.

Millennium, says Tepper, has taken combinatorial chemistry a step further to become an expert in testing the multitudinous products of combinatorial chemistry reactions with enzyme targets in a technique called 'high-throughput screening.'

"If you're dealing with 10 or 96 samples at a time, that's doable by one person with a pipettor," explains Tepper. "When you're dealing with sets of tens of thousands of chemical compounds, automation becomes quite necessary."

The result of automation, he claims is both a reduction in expenses and a boost in the efficiency of scientists. "When you free up individuals to be creative and do the work that can't be done by machines, you free up a lot of potential," he says.

Renee J. Raphael, Joshua L. Kwan, Eran K. Mukamel and Amita Mi. Shukla contributed to the reporting of this story.

"When you free up individuals to be creative... you free up a lot of potential."  Robert Tepper, M.D.

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