Compounds and methods for reducing ATXN3 expression

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United States of America Patent

PATENT NO 11583548
APP PUB NO 20190247420A1
SERIAL NO

16344254

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ATTORNEY / AGENT: (SPONSORED)

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Abstract

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Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 mRNA in a cell or animal, and in certain instances reducing the amount of Ataxin-3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to prevent or ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include SCA3.

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Patent Owner(s)

  • IONIS PHARMACEUTICALS, INC.

International Classification(s)

Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Freier, Susan M San Diego, US 509 6151

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