AGENTS FOR RNAi, COMPOSITIONS AND METHODS OF THEIR USE FOR THE TREATMENT OF DISEASES ASSOCIATED WITH TRANSTHYRETIN (TTR) Russian patent published in 2023 - IPC C07H21/00 C12N15/113 A61K31/7088 A61K31/713 

Abstract RU 2804776 C2

FIELD: biotechnology.

SUBSTANCE: group of inventions is described, including a double-stranded RNAi agent for inhibiting the expression of transthyretin (TTR) in a cell (variants), a pharmaceutical composition for inhibiting the expression of transthyretin (TTR), a method of inhibiting the expression of transthyretin (TTR) in a cell and a set for its implementation, a method of treating or preventing a TTR-associated disease in an individual, and a set for its implementation. In one embodiment, the double-stranded agent comprises a sense strand and an antisense strand with the following sequences of SEQ ID NO: 2211 and 2217, respectively.

EFFECT: invention expands the arsenal of agents for inhibiting the expression of transthyretin (TTR) in the cell.

58 cl, 15 dwg, 10 tbl, 51 ex

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RU 2 804 776 C2

Authors

Rajeev, Kallanthottathil, G.

Zimmermann, Tracy

Manoharan, Muthiah

Maier, Martin

Kuchimanchi, Satyanarayana

Charisse, Klaus

Dates

2023-10-05Published

2012-11-16Filed