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...
07:00 , Oct 16, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Hyperoxaluria Alanine-glyoxylate aminotransferase (AGXT) In vitro studies suggest dequalinium chloride (DECA) could help treat primary hyperoxaluria (PH1), a disease caused by AGXT mutations. AGXT...
07:00 , Apr 30, 2012 |  BC Week In Review  |  Clinical News

AMT-030 regulatory update

The European Commission granted Orphan Drug designation for AMT-030 from uniQure to treat primary hyperoxaluria type 1. AMT-030 is in preclinical testing, with clinical testing expected to start in 3 years. The product is an...