The Gatekeeper 

What if scientists devised a strategy to tackle some of the world's most notorious diseases, but just one company held all the patents?

Page 6 of 6

The push may come, in fact, from the Dow AgroSciences agreement, which gives Dow the right to option the products of its research for an exclusive commercial license. If that should happen, Sangamo would have to find a way to share some of its secrets. It already has been preparing for that eventuality by improving the speed and automation of zinc finger production, so that it can be handed over more easily. "We will come to some agreement, whatever makes commercial sense, so that the making of zinc finger proteins is not a bottleneck," Wolffe promises. And indeed, Wolffe agrees that such a small company will need collaborators in order to expand the number of diseases it can investigate. It also will need partners to help get its current test products through the final, much larger, stages of clinical testing.

The next few years should be make-or-break ones for zinc finger therapeutics, as more of Sangamo's research moves from the lab to human trials. In addition to the HIV and diabetes-related treatments, Sangamo is considering its possibilities for cancer, Parkinson's, peripheral artery disease, X-SCID, and sickle cell anemia.

If the history of gene therapy is anything to go on, Friedmann warns, zinc finger therapeutics won't turn up in your local doctor's office anytime soon — there are still many kinks to be worked out. "Delivery will be difficult, efficiency will be difficult, and the body will try very hard to figure out ways to get around the manipulation that is being performed, so there will be a lot of surprises and no quick cures," he said. But he's quick to add that the field is eagerly watching: "This technology is really enticing because it's really the first time one begins to get a glimmer of how to correct the gene," he said.

As for Sangamo's top exec, it's hard for him to talk about the future without sounding a little giddy. "If we are successful, it will be the creation of the first new medical platform in the post-genomic era," Lanphier said. "If that doesn't get you excited, nothing will."

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