SiRNAs with vinylphosphonate at the 5′ end of the antisense strand

US11560563B2 · kind B2 · utility

Assignee

• Sapience Therapeutics, Inc. · DE

Inventors

• Adrien Weingärtner · Berlin, DE
• Lucas Bethge · Potsdam, DE

Key dates

• Filing date: Apr 5, 2019
• Grant date: Jan 24, 2023
• Expiry date: Jul 24, 2039

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/51
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to nucleic acids for inhibiting expression of a target gene in a cell, comprising at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene to be inhibited. The first strand of the nucleic acid has a terminal 5′ (E)-vinylphosphonate nucleotide that is linked to the second nucleotide in the first strand by a phosphodiester linkage.