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(19)

RU

(11)

2 782 034

(13)

C2

(51)

IPC

C07H21/00(2006-01-01)

A61K31/7125(2006-01-01)

A61P9/00(2006-01-01)

(21) (22)

Application

2018136140, 2014-05-01

(24)

Start date

2014-05-01

(22)

Actual filing date

2014-05-01

(45)

Published

2022-10-21

(72)

Inventor

Prakash Tkhazha P.Set Punit P.Svejz Erik E.

(73)

Holder

Ionis Farmasyutikalz, Ink.

COMPOSITIONS AND METHODS FOR MODULATION OF HBV AND TTR EXPRESSION Russian patent published in 2022 - IPC C07H21/00 A61K31/7125 A61P9/00 

Abstract RU 2782034 C2

FIELD: pharmacology; medicine.

SUBSTANCE: group of inventions relates to the field of pharmacology and medicine, and it is aimed at the treatment of transthyretin amyloidosis. An oligomeric compound is disclosed, while an anionic form of the oligomeric compound is presented by the following chemical structure (SEQ ID NO: 12). In addition, a form of free acid, sodium salt of the above-mentioned oligomeric compound, and an oligomeric compound containing modified oligonucleotide and a conjugating group, characterized by the following formula (SEQ ID NO: 12), are disclosed. A pharmaceutical composition for the treatment of transthyretin amyloidosis, based on the above-mentioned compounds, the use of presented compounds in the production of a drug for the treatment of transthyretin amyloidosis, and, in addition, a method for the treatment of transthyretin amyloidosis in a subject, including administration to the subject of therapeutically effective amount of the oligomeric compound or the pharmaceutical composition containing the specified oligomeric compound, are also disclosed.

EFFECT: group of inventions provides effective treatment of transthyretin amyloidosis.

15 cl, 108 tbl, 113 ex

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RU 2 782 034 C2

Authors

Prakash Tkhazha P.

Set Punit P.

Svejz Erik E.

Dates

2022-10-21—Published

2014-05-01—Filed

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