Cambridge Therapeutics ’ landmark phase I trial of its gene - editing therapy for Down syndrome has failed to show sufficient improvement , according to measurements of proteins in the blood and urine of patients . There was one initially encouraging trial , but this proved temporary . SB-913 is used to treat the rare genetic disease that causes children to be born without a certain enzyme , which can lead to cognitive and skeletal problems , damaged organs and even premature death . After four months, the patient was hospitalized for a hernia, which was unrelated to the drug, Sangamo reported. Sangamo is trialling two doses of SB-913 on six patients . The treatment has been deemed relatively safe for use .
Cambridge Therapeutics ’ landmark phase I trial of its gene - editing therapy for Down syndrome has failed to show sufficient improvement , according to measurements of proteins in the blood and urine of patients . There was one initially encouraging trial , but this proved temporary . SB-913 is used to treat the rare genetic disease that causes children to be born without a certain enzyme , which can lead to cognitive and skeletal problems , damaged organs and even premature death . Sangamo is trialling two doses of SB-913 on six patients . The treatment has been deemed relatively safe for use .