BC Innovations | Jan 16, 2019
Distillery Therapeutics


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...
BC Innovations | Oct 16, 2014
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...
BC Week In Review | Apr 30, 2012
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...
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