19:00 , Jan 4, 2018 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: December 2017

New Therapeutic Targets and Biomarkers: December 2017...
01:09 , Dec 13, 2017 |  BC Innovations  |  Distillery Therapeutics

Endocrine / Metabolic

INDICATION: Pompe's disease Mouse studies suggest gene therapies delivering secretion-optimized GAA variants could help treat Pompe’s disease. The gene therapies consist of a liver-tropic adeno-associated viral serotype 8 (AAV8) vector encoding one of two GAA...