Short antisense compounds with gapmer configuration
US9045754B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | May 7, 2007 |
| Grant date | Jun 2, 2015 |
| Priority date | — |
| Expiry date | Aug 4, 2030 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/3515
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.