By Daniel Grushkin

Researchers hoping to write the next chapter of biomedicine know that their progress will hinge largely on their ability to edit genes — cutting out unwanted DNA and manipulating the sequences they want to keep. Acolytes of gene-editing technology have recently been encouraged by early data about Sangamo BioSciences’ new zinc finger nuclease–based therapies. In March, the California biotech reported preliminary phase 1 trial results showing that its HIV treatment, which uses editing to turn off the gene for the receptor on the cell membrane that the virus exploits, was safe and effective at improving people’s T cell counts. Two months later, Sangamo scientists presented additional evidence at the American Society of Gene & Cell Therapy meeting in Seattle demonstrating in mouse and cell models the technology’s promise in a number of other diseases, including hemophilia, so-called ‘bubble boy disease’ and a form of acquired blindness.

Sangamo’s success has fed a growing enthusiasm for genome engineering technologies. And now, a handful of biotechs with competing technologies and business models are vying to be the go-to company for gene-editing applications. But not all technologies are created equal, and there’s debate within the research community about which technique is best for which purpose. “It’s a bit like ‘which one will win out — PCs or Macs?’,” says Bert Vogelstein, a cancer researcher at the Johns Hopkins University School of Medicine in Baltimore. “The one people will choose is the one that’s available, easiest, cheapest and the one they have experience with.”

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