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Sangamo Genome Editing Trial, Assessing MPS II Candidate, Treats First Patient

06:06 EST 15 Nov 2017 | Genetic Engineering News

Sangamo Therapeutics said today it has treated the first patient in the Phase I/II CHAMPIONS clinical trial assessing its in vivo genome-editing candidate therapy SB-913 for mucopolysaccharidosis type II (MPS II), also called Hunter syndrome. “We are at the start of a new frontier of genomic medicine,” declared Sangamo CEO Sandy Macrae, M.B., Ch.B., Ph.D., in a statement. “For the first time, a patient has received a therapy intended to precisely edit the DNA of cells directly inside the body.” That patient was 44-year-old Brian Madeux, who told the Associated Press he found being the trial’s first patient “kind of humbling” and added: “I’m willing to take that risk. Hopefully it will help me and other people.” SB-913 uses Sangamo's zinc finger nuclease (ZFN) genome-editing technology to treat MPS II by inserting a corrective gene into a precise location in the DNA of liver cells. ...

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