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. The first U.S. trial of a gene-editing medicine in humans isn’t going well. 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. 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.