When Genes Come True

“We did it!” shouted T-shirts sported by the twenty-something technicians in the hallways of Human Genome Sciences (HGS) at a ribbon-cutting ceremony this spring. The staff at the Rockville, Md., biotech company was celebrating the opening of a $42 million manufacturing plant that marks the firm’s evolution from research startup to an aspiring drug maker.

The event is also a watershed in the emerging era of human “genomics”-the large-scale study of man’s estimated 80,000 to 100,000 genes. HGS helped kick off the commercialization of the field in 1992, when it was founded with plans to use scores of automated DNA sequencing machines to rapidly decode genes. Now, $275 million later, HGS is taking the first batch of drugs discovered via genomics methods into human testing.

Before genomics, the path to a biotech drug typically began when scientists identified the gene that coded for an important, well-studied protein, such as insulin. With the genetic recipe in hand, they could use recombinant DNA techniques to genetically engineer microbes used in large-scale manufacturing of the protein.

But finding a protein’s gene often was a painstaking enterprise that took years of effort. So, HGS and other genomics companies decided to reverse the process by decoding human DNA on a massive scale. They then could fish out genes and work forward, biochemically speaking, to discover new proteins that are involved in disease processes.

It’s a rapid-fire approach that William Haseltine, HGS’ CEO, has long said would produce a new pharmacopeia. Starting last year, the company began making good on the promise, introducing several candidates into early-stage human clinical trials, including proteins for protecting bone-marrow cells in chemotherapy patients and for facilitating wound healing.

The technology is also identifying hormones and enzymes that conventional drugs can target. Genomics is responsible for more than half the therapeutic targets that pharmaceutical giant Bristol-Myers Squibb in Princeton, N.J., now has in its sights, according to director of science and technology Ron Peppin.

A costly, uncertain road lies ahead before any blockbuster with an HGS logo appears on pharmacy shelves, however. Less than one-quarter of drugs pan out in clinical trials, a process that can easily cost more than $100 million and last a decade.