Voyager Therapeutics announces VY-SOD101 data at ASGCT
Voyager Therapeutics announced new data presentations at the American Society of Gene and Cell Therapy taking place May 16-19, 2018, in Chicago, Ill. Voyager presented results for VY-SOD101, which targets the superoxide dismutase 1 gene, the first mutant gene discovered to be causal for the development of amyotrophic lateral sclerosis. The results demonstrated that a one-time administration of VY-SOD101 lowered SOD1 mRNA levels by 78% in the spinal cord motor neurons of non-human primates. Additional new data at this year's ASGCT meeting included tolerability data in non-human primates for VY-HTT01 for Huntington's disease, along with previous data with VY-HTT01 that demonstrated a 54% suppression of huntingtin mRNA in the non-human primate putamen after a single administration. Presentations at ASGCT also included comparability data between Voyager's baculovirus/Sf9 and HEK293 triple transfection manufacturing systems.