Nine things to know about Sangamo Therapeutics and its first-in-human genome editing study

The first clinical trial using genome editing to treat real patients is nearing an early but crucially important data reveal.

Until now, the only data available involving the removal or repair of disease-causing DNA have come from experiments in test tubes and animals. But late last year, the biotech company Sangamo Therapeutics started a clinical trial using a one-time, genome-editing fix to treat people born with a rare, inherited disease.

This is still underway, but Sangamo intends to present preliminary results at a medical conference on Sept. 5.

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