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Shares of Sangamo Therapeutics Inc. fell 13% on Wednesday after results of a half-life trial of its gene-editing candidate SB-913 for the rare metabolic syndrome of Hunter's Syndrome . Two patients participating in the clinical trial had reductions in a key biomarker of the disease at 16 weeks, the company said. Dr. Joseph Muenzer, professor of pediatrics and professor of pediatrics genetics at the University of North Carolina School of Medicine at Chapel Hill and senior researcher on the study, said in a statement. communicated. The trial, called "Champions", recruited two patients in three groups, ranging from low to medium doses and high. The company touted the results of two patients in the mid-dose group of treatment; The listing and dosing of patients at the high dose was simply completed, he said. Hunter syndrome, also known as MPS II, mainly affects boys and causes progressive cell damage. The Sangamo gene editing candidate aims to treat Hunter's syndrome by adding a new copy of a gene in the DNA of a patient's liver cells. This process was performed in vivo, or in the body, marking the first such experiment in humans, the company said; In other types of gene editing and gene therapies, the process takes place outside the body. SB-913 was generally well tolerated, according to Sangamo; Although two serious side effects were observed, the investigator decided that they were primarily related to the disease and not to the treatment. The company's shares jumped 13.7% in the last three months to $ 19.05, up from a 5.4% increase in the S & P 500 and 4.7% in the Dow Jones Industrial Average.
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