17:10 , Jan 16, 2019 |  BC Innovations  |  Distillery Therapeutics

Endocrine/metabolic

INDICATION: Hyperoxaluria Mouse studies suggest CRISPR-Cas9-based gene editing therapies targeting HAO1 could help treat primary hyperoxaluria type 1, which is caused by loss-of-function AGXT mutations and results in renal accumulation of glyoxalate and oxalate. The...
18:15 , Feb 28, 2018 |  BC Innovations  |  Distillery Techniques

Drug platforms

TECHNOLOGY: Nucleic acids Modifying the seed region of hepatocyte-targeted siRNAs could help develop therapies with minimal hepatic toxicity. The approach involves generating siRNA guide strands containing a glycol nucleic acid (GNA) nucleotide in the seed...
07:00 , May 23, 2016 |  BC Week In Review  |  Clinical News

DCR-PH1: Phase I started

Dicerna began the open-label, dose-escalation, international Phase I DCR-PH1-101 trial to evaluate IV DCR-PH1 in patients ages >=12. DCR-PH1 uses lipid nanoparticle (LNP) delivery technology from Arbutus (see BioCentury, Jan. 5, 2015). Arbutus Biopharma Corp....
08:00 , Jan 5, 2015 |  BC Week In Review  |  Company News

Tekmira, Dicerna deal

Tekmira granted Dicerna worldwide rights to lipid nanoparticle (LNP) technology to deliver Dicerna’s DCR-PH1 . The product is a dicer substrate short interfering RNA targeting mRNA encoded by the hydroxyacid oxidase 1 ( HAO1 )...