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Sangamo Data Is A Moment For Gene Editing, But Leaves Questions

 
• By Jessica Merrill

Sangamo announced the first human data for a gene editing treatment, SB-913 for MPS II, but the early data in just four patients shows gene editing technology has a long way to go before new medicines reach the market.

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Sangamo Therapeutics Inc. crossed an important milestone Sept. 5, becoming the first drug company to release efficacy data on a gene editing therapy in human patients. The data were early and in only four patients, but Sangamo was able to show a benefit on a key biomarker of the disease being studied.

The data also raised questions, however, because it didn't show the medicine helped patients produce a critical missing enzyme, and it remains unclear what

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