BioCentury | Oct 24, 2020
Translation in Brief

UNC Chapel Hill Angelman gene therapy rescues motor function; plus data backing planned Pacylex, Kronos clinical trials and more

...Clinic and University College London researchers has described in Science Translational Medicine an immunoassay that detects polyglutamine ATXN3...
...healthy individuals. They also found a strong link between the disease-causing DNA repeat expansion in ATXN3...
...Alberta Kronos Bio Inc. Massachusetts Institute of Technology Mayo Clinic University College London Ohana Biosciences Ubiquitin protein ligase E3A (UBE3A) (E6AP) Ataxin 3 (ATXN3) Cyclin...
BioCentury | Dec 6, 2018
Translation in Brief

New Therapeutic Targets and Biomarkers: November 2018

New Therapeutic Targets and Biomarkers: November 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during November. Therapeutic targets are defined as any protein, gene or other molecule...
BioCentury | Nov 13, 2018
Distillery Therapeutics

Ophthalmic disease

INDICATION: Blindness Mouse studies suggest inhibiting ATXN7 could help treat vision loss in spinocerebellar ataxia type 7 (SCA7), which is caused by gain-of-function mutations in the gene. In a genetic mouse model of SCA7, intravitreal...
BioCentury | Mar 7, 2018
Distillery Therapeutics

Neurology

...a mouse embryonic fibroblast cell line expressing mutant ATXN3, knockout of KPNA3 decreased levels of ATXN3...
...and testing KPNA3 inhibitors in models of SCA3. TARGET/MARKER/PATHWAY: Karyopherin subunit α 3 (KPNA3); ataxin 3 (ATXN3...
...2018 doi:10.1073/pnas.1716071115 CONTACT: Thorsten Schmidt, University of Tübingen, Tübingen, Germany email: thorsten.schmidt@med.uni-tuebingen.de Hongjiang Li University of Tuebingen Ataxin 3 (ATXN3) Karyopherin...
BioCentury | Feb 23, 2018
Company News

Takeda, Wave in CNS nucleic acid deal

...WVE-120101, WVE-120102 and WVE-3972-01. Takeda also has the option for a preclinical program targeting ataxin 3 (ATXN3...
...Pharmaceutical Co. Ltd. (Tokyo:4502), Osaka, Japan Business: Neurology Chris Lieu WVE-120101 WVE-120102 Takeda Pharmaceutical Co. Ltd. Wave Life Sciences Ltd. Ataxin 3 (ATXN3) Chromosome...
BioCentury | Feb 21, 2018
Company News

Takeda, Wave in CNS nucleic acid deal

...WVE-120101, WVE-120102 and WVE-3972-01. Takeda also has the option for a preclinical program targeting ataxin 3 (ATXN3...
...today's announcement as a "sell the news" opportunity. Chris Lieu WVE-120101 WVE-120102 Takeda Pharmaceutical Co. Ltd. Wave Life Sciences Ltd. Ataxin 3 (ATXN3) Chromosome...
BioCentury | May 11, 2017
Translation in Brief

Attackin' Ataxin

Two independent academic groups have shown different antisense oligos (ASOs) made by Ionis Pharmaceuticals Inc. (NASDAQ:IONS) targeting the same protein, ataxin 2 (ATXN2), extended survival and improved motor function in models of amyotrophic lateral sclerosis...
BioCentury | May 4, 2017
Targets & Mechanisms

The polyQ diet

...other proteins directly regulate the autophagy-inducing protein BECN1. (1) Wild-type ATXN3. The non-mutant form of ATXN3...
...normal conditions, the polyQ domain of ATXN3 binds BECN1 , after which other sites on ATXN3...
...of BECN1. This also enables BECN1 degradation to reduce autophagy and cause neurodegeneration. ATXN3 - Ataxin 3...
BioCentury | Apr 25, 2017
Distillery Therapeutics

Neurology

INDICATION: Ataxia; amyotrophic lateral sclerosis (ALS) Mouse studies suggest inhibiting ATXN2 expression could help treat spinocerebellar ataxia type 2 (SCA2), and ALS and other TDP-43 -associated neurodegenerative diseases. In two transgenic mouse models of SCA2...
BioCentury | Aug 11, 2016
Distillery Therapeutics

Therapeutics: Ataxin 2 (ATXN2; SCA2)

Endocrine/metabolic disease INDICATION: Obesity Caenorhabditis elegans studies suggest promoting ATXN2 expression could help treat obesity. In C. elegans on a normal diet, overexpression of the worm ortholog of human ATXN2 decreased body size and fat...
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