Positive Topline Results Released from Study of Gene Therapy in OTC Deficiency
March 7th 2018Ultragenyx Pharmaceutical, Inc. released positive long-term safety and efficacy data from the first dose cohort of the Phase 1/2 study of DTX301. The investigational AAV gene therapy, is intended for the treatment of OTC deficiency.
CRISPR Used to Disable Defective Gene in ALS
January 4th 2018Recent data have found that the same gene editing platform can disable the defective gene responsible for amyotrophic lateral sclerosis in mice. The therapy, which extended the lifespan of the mouse models by 25%, delayed the onset of the muscle wasting which characterizes the disease.