00:50 , Mar 7, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Ataxia Cell culture, fruit fly and mouse studies suggest inhibiting KPNA3 could help treat spinocerebellar ataxia type 3 (SCA3), which is caused by mutant ATXN3 containing polyglutamine repeats that accumulates in the nucleus. In...
15:23 , Feb 23, 2018 |  BC Week In Review  |  Company News

Takeda, Wave in CNS nucleic acid deal

Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) and Wave Life Sciences Ltd. (NASDAQ:WVE) partnered to develop and commercialize nucleic acid therapies to treat CNS diseases. Wave will receive an upfront payment of $170 million, including $110 million...
00:15 , Feb 21, 2018 |  BC Extra  |  Company News

Takeda, Wave in CNS nucleic acid deal

Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) and Wave Life Sciences Ltd. (NASDAQ:WVE) partnered to develop and commercialize nucleic acid therapies to treat CNS diseases. Wave will receive an upfront payment of $170 million, including $110 million...
22:49 , May 4, 2017 |  BC Innovations  |  Targets & Mechanisms

The polyQ diet

Expanded polyglutamine repeats at the ends of disease-causing proteins are well known to promote formation of aggregates that drive various neurodegenerative disorders. A new study from the University of Cambridge shows those repeats have a...
07:00 , May 26, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Sirtuin 1 (SIRT1); ataxin 3 (ATXN3)

Neurology INDICATION: Ataxia Patient sample and mouse studies suggest promoting SIRT1 activity could help treat spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disease caused by mutant ATXN3 encoding polyglutamine repeats. Levels of SIRT1 were lower...
07:00 , Oct 13, 2011 |  BC Innovations  |  Distillery Therapeutics

Indication: Neurology

Indication Target/marker/pathway Summary Licensing status Publication and contact information Neurology Huntington's disease (HD) Huntingtin (HTT); ataxin 3 (ATXN3) In vitro and mouse studies identified peptoids that bind polyglutamate repeats and could help treat HD. A...
07:00 , Aug 6, 2009 |  BC Innovations  |  Tools & Techniques

Targeting toxic RNA

Researchers at the University of Rochester have used an antisense molecule to reverse muscular defects in mice with myotonic dystrophy type 1. 1 The approach represents a new way to attack the most common form...
07:00 , Jul 16, 2009 |  BC Innovations  |  Distillery Therapeutics

Indication: Various

Various Muscular dystrophy; ataxia Dystrophia myotonica protein kinase (DMPK); ataxin 3 (ATXN3); muscleblind-like 1 (MBNL1) In vitro studies identified inhibitors of RNA-protein interactions that could help treat myotonic dystrophy type 1 (DM1) and spinocellular ataxia...
07:00 , May 28, 2009 |  BC Innovations  |  Distillery Therapeutics

Indication: Neurology

This week in therapeutics Indication Target/marker/ pathway Summary Licensing status Publication and contact information Neurology Huntington's disease (HD); Machado-Joseph disease (MJD) Huntingtin (HTT); Ataxin 3 (ATXN3) Studies in human and mouse cell culture suggest that...