Modified double-stranded RNA agents
US12385043B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Aug 23, 2024 |
| Grant date | Aug 12, 2025 |
| Priority date | — |
| Expiry date | Aug 23, 2044 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/52
- 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 sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.