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First attempt at genome editing in U.S. patients produces disappointing — and sobering — results

  • Adam Feuerstein

By Adam Feuerstein Feb. 7, 2019

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Sangamo zinc fingers illo
Alex Hogan/STAT

The first attempt at using genome editing to treat and cure patients with a rare, inherited disease has produced disappointing results in a small clinical trial.

Despite the setback, Sangamo Therapeutics (SGMO), the biotech developing the genome-editing therapy, intends to keep trying. Transient improvements observed in a single patient suggest a more potent version of its treatment — already being readied for use — might be more effective, the company said.

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About the Author Reprints

Adam Feuerstein

Adam Feuerstein

Senior Writer, Biotech

Adam is STAT’s national biotech columnist, reporting on the intersection of biotech and Wall Street. He's also a co-host of "The Readout LOUD" podcast.

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  • Hi Adam, I’m a great fan of yours!
    Thanks for the summary, but I wonder what is your opinion? Based on the observed data, do you think it could be solved by dosing 1e14 / using ZFN2? Or more luck in children vs. adults? Simply to early to judge anything? Thanks for giving your take!

    Cheers,
    Dan

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