Hunter ’ landmark phase I trial of its gene-editing treatment for Sangamo Therapeutics syndrome has failed to show sufficient improvement, according to measurements of proteins in the blood and urine of patients. Xconomy San Francisco — 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. Brisbane, CA is trialling six doses of SB-913 on three patients. The treatment has been deemed relatively safe for use.
Hunter ’ landmark phase I trial of its gene-editing treatment for Sangamo Therapeutics 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. Brisbane, CA is trialling six doses of SB-913 on three patients. The treatment has been deemed relatively safe for use.