The first U.S. trial of a gene-editing medicine in humans isn’t going well. Sangamo Therapeutics (NASDAQ: SGMO) reported Phase 1 results this morning for its treatment for Hunter’s syndrome, a rare genetic disease. While relatively safe, the treatment, known as SB-913, did not seem to help patients much, based on measurements of proteins in their blood and urine that are meant to show that the treatment is working.
Sangamo shares were down more than 35 percent, to $7.75, in midday trading.
The study, known as CHAMPIONS, is testing three doses of SB-913 across six patients. It’s a tiny sample size,… Read more »
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