Modified RNA agents with reduced off-target effect
US12247203B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | May 16, 2019 |
| Grant date | Mar 11, 2025 |
| Priority date | — |
| Expiry date | Apr 12, 2042 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/53
- WIPO fieldBiotechnology
- WIPO sectorChemistry
Abstract
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2′-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.