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Last Updated: November 27, 2020

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Claims for Patent: 9,061,044

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Summary for Patent: 9,061,044
Title:Modulation of transthyretin expression
Abstract: Provided herein are methods, compounds, and compositions for reducing expression of transthyretin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate transthyretin amyloidosis, or a symptom thereof.
Inventor(s): Freier; Susan M. (San Diego, CA)
Assignee: Isis Pharmaceuticals, Inc. (Carlsbad, CA)
Application Number:14/184,984
Patent Claims: 1. A compound consisting of a pharmaceutically acceptable salt of a single-stranded modified oligonucleotide consisting of 20 linked nucleosides having a nucleobase sequence consisting of SEQ ID NO: 80, wherein the modified oligonucleotide comprises: a gap segment consisting often linked deoxynucleosides; a 5' wing segment consisting of five linked nucleosides; and a 3' wing segment consisting of five linked nucleosides; wherein the gap segment is positioned between the 5' wing segment and the 3' wing segment, wherein each nucleoside of each wing segment comprises a 2'-O-methoxyethyl sugar, wherein each internucleoside linkage is a phosphorothioate linkage, and wherein each cytosine of the modified oligonucleotide is a 5-methylcytosine.

2. The compound of claim 1, wherein the pharmaceutically acceptable salt is a sodium salt.

3. The compound of claim 1, wherein the pharmaceutically acceptable salt is a potassium salt.

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Serving leading biopharmaceutical companies globally:

Merck
Dow
McKinsey
AstraZeneca
Harvard Business School
Johnson and Johnson

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