22:22 , Nov 1, 2018 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: October 2018

New Therapeutic Targets and Biomarkers: October 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during October. Therapeutic targets are defined as any protein, gene or other molecule...
18:41 , Oct 18, 2018 |  BC Innovations  |  Distillery Therapeutics

Endocrine/Metabolic; neurology

INDICATION: Porphyria; pain Mouse, rabbit and monkey studies suggest a lipid nanoparticle (LNP)-encapsulated mRNA encoding HMBS could help treat acute intermittent porphyria (AIP), which is caused by hepatic HMBS deficiency, and AIP-associated pain. In mouse...