Sangamo, With a Lock on Genetic Switch Technology, Seeks to Morph Into Drugmaker

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a mid-stage trial in June at the American Diabetes Association. The trial, among patients with mild to moderate diabetic neuropathy, showed that the Sangamo drug could improve nerve regrowth and blood vessel growth after doctors took a biopsy from the patient. Patients also showed an improvement in nerve conduction velocity, a key measurement of nerve damage. “Within the diabetic neuropathy community, there’s an incredible amount of enthusiasm,” Lanphier says.

This is the kind of data that doctors will want to see—a connection between what’s happening at the molecular level and an improvement in clinical symptoms of disease. But before anyone jumps for joy, Sangamo needs to generate data from an ongoing study of 150 patients that compares SB-509 directly to a placebo, after six months. That study, enrolling patients with moderate to severe diabetic neuropathy, is expected to provide some answers in the second half of 2011, Lanphier says.

Sangamo hasn’t bet the whole company on diabetic neuropathy. It is expected to release some data from a treatment for amyotrophic lateral sclerosis (Lou Gehrig’s disease) next month at The Society for Neuroscience meeting. Sangamo also has an experimental HIV treatment it is developing with researchers at UCLA and the University of Pennsylvania, supported by a $14.5 million grant from the California Institute of Regenerative Medicine.

Not many on the Street are likely to pay much attention to these programs, even if they show promise at an early stage. More cash and credibility from big-name partners wouldn’t hurt, but that still probably won’t propel Sangamo to the top of the big fund managers’ radar screens. To hear Lanphier, true rewards will come when Sangamo generates the kind of compelling evidence from late-stage clinical trials that shows his company really owns a blockbuster drug. That doesn’t happen overnight, and all he can do for now is keep focused on the fundamentals he thinks it takes to get there.

“I don’t get up in the morning and say ‘Gosh, the stock’s here, what do I do?’ I ask things like ‘Are we working on the right things? Do we have the right people? Do we have the capital to get there?’ That’s day-to-day, hour-to-hour,” Lanphier says. “You don’t go into Sangamo for quarter-to-quarter earnings reports.”

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