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

New Therapeutic Targets and Biomarkers: December 2017

New Therapeutic Targets and Biomarkers: December 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during December 2017. Therapeutic targets are defined as any protein, gene or other molecule...
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 variants...