Sangamo Therapeutics’ landmark phase I trial of its gene-editing treatment for Hunter syndrome has failed to show sufficient improvement, according to measurements of proteins in the blood and urine of patients. There was one initially encouraging response, but this proved temporary. The results are a setback for the company, which had hoped that the study would be completed by early next year. Sangamo is trialling three doses of SB-913 on six patients. The treatment has been deemed relatively safe for use.
Sangamo Therapeutics’ landmark phase I trial of its gene-editing treatment for Hunter syndrome has failed to show sufficient improvement, according to measurements of proteins in the blood and urine of patients. There was one initially encouraging response, but this proved temporary. SB-913 is intended 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 three doses of SB-913 on six patients. The treatment has been deemed relatively safe for use.