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

...Albert La Spada, Duke University School of Medicine, Durham, N.C. email: al.laspada@duke.edu Chris Lieu Duke University Ionis Pharmaceuticals Inc. Ataxin 7 (ATXN7) (SCA7) Blindness...
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 | 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...
BioCentury | Nov 8, 2012
Distillery Therapeutics

Indication: Neurology

Indication Target/marker/pathway Summary Licensing status Publication and contact information Neurology Ataxia Potassium channel KCa2.2 (KCNN2); KCNN3; ataxin 2 (ATXN2; SCA2) Mouse studies suggest positive allosteric modulators (PAMs) of KCNN2 could help treat spinocerebellar ataxia type...
BioCentury | Sep 16, 2010
Targets & Mechanisms

Aggregation party in ALS

Aggregation and mislocalization of TDP-43 are at the heart of amyotrophic lateral sclerosis, but it is difficult to manipulate the protein directly because of its broad role in cellular survival. University of Pennsylvania researchers now...
BioCentury | Sep 9, 2010
Distillery Therapeutics

Indication: Neurology

Indication Target/marker/ pathway Summary Licensing status Publication and contact information Neurology Amyotrophic lateral sclerosis (ALS) Ataxin 2 (ATXN2; SCA2); TAR DNA binding protein 43 (TDP-43; TARDBP) A study in yeast, flies and ALS patients suggests...
BioCentury | Jul 30, 2009
Distillery Therapeutics

Indication: Neurology

Indication Target/marker/pathway Summary Licensing status Publication and contact information Neurology Ataxia Ataxin 2 (ATXN2; SCA2) Studies in mice suggest that dantrolene could help treat spinocerebellar ataxia type 2, a neurodegenerative disease caused by polyglutamine expansion...
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