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Sangamo's landmark genome editing trial gets mixed reception

Nature Biotechnology volume 36, pages 907–908 (2018)Cite this article

Sangamo Therapeutics' preliminary results from a trial of its first in vivo zinc finger nuclease genome editing therapy suggest that its mucopolysaccharidosis II (MPS II) treatment is safe and may yet prove to be effective. The early-stage data on SB-913, disclosed on September 5, were not sufficiently convincing for many investors, however, who drove down its share price by over 30%. Other genome editing companies, pursuing completely different approaches, were also caught up in the sell-off. As the first therapies based on the CRISPR-Cas9 system near the clinic (Table 1), investor skittishness is likely to increase further.

Table 1 Selected genome editing programs in human testing or preparing for trials
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    Cormac Sheridan

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Sheridan, C. Sangamo's landmark genome editing trial gets mixed reception. Nat Biotechnol 36, 907–908 (2018). https://doi.org/10.1038/nbt1018-907

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