Abstract: The present invention provides an oligonucleotide, containing a compound of formula (Ia), or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof. The compound of formula (I) is contained inside the oligonucleotide or at the 5? terminal and/or the 3? terminal, and is used for improving the ability of double-stranded RNA to pass through a cell membrane. The present invention also relates to a double-stranded RNA, a cell, a pharmaceutical composition and a kit containing the oligonucleotide.
Type:
Application
Filed:
October 30, 2023
Publication date:
June 25, 2026
Applicant:
Rona Bioscience, Limited
Inventors:
Jinyu Huang, Hao Zou, Junkai Liu, Yang Ke
Abstract: The present invention provides a double-stranded RNA comprising a nucleotide analog. The double-stranded RNA of the present invention exhibits one or more of enhanced stability, reduced off-target toxicity, and enhanced effectiveness.
Abstract: The present invention provides an oligonucleotide having a cyclic phosphonate modification. The oligonucleotide of the present invention exhibits one or more among enhanced stability, reduced off-target toxicity, and enhanced effectiveness.
Abstract: Provided in the present invention is a ligand containing N-acetylgalactosamine, wherein the ligand contains a conjugated group as represented by formula (X?) and a nucleic acid molecule containing the targeting ligand.
Abstract: Provided in the present invention is a double stranded RNA having a nucleotide analog. The double-stranded RNA of the present invention shows one or more of enhanced stability reduced off-target toxicity and enhanced efficiency.