Sangamo Therapeutics Inc. SGMO, -21.00% shares dropped 13% in Wednesday premarket trade after the company released results for a phase 1/2 trial of its gene editing candidate SB-913, which is intended for the rare metabolic disorder Hunter syndrome. Two patients in the clinical trial had reductions of a key biomarker of the disease at 16 weeks, the company said. That and other reductions seen were “encouraging” but the company will need to withdraw enzyme replacement therapy, which is used to manage the disease’s symptoms, to “understand the clinical relevance of these changes,” Dr. Joseph Muenzer, a professor of pediatrics and genetics at the University of North Carolina School of Medicine in Chapel Hill and principal investigator on the study, said in a statement. The trial, called “Champions,” enrolled two patients in three groups, ranging from low to mid and high doses. The company touted the results of two patients in the group on a mid dose of the therapy; enrollment and dosing of patients on the high dose was just completed, it said. Hunter syndrome, also called MPS II, primarily affects boys and causes progressive cell damage. Sangamo’s gene editing candidate aims to treat Hunter syndrome by adding a new copy of a gene into the DNA of a patient’s liver cells. This process was done 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 is completed outside of the body. SB-913 was generally well-tolerated, according to Sangamo; though two serious side effects were seen, the investigator decided they were primarily related to the disease and not the treatment. Company shares have surged 13.7% over the last three months to $19.05, compared with a 5.4% rise in the S&P 500 SPX, -0.56% and a 4.7% rise in the Dow Jones Industrial AverageDJIA, -0.15%
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