Single-stranded RNAi agents containing an internal, non-nucleic acid spacer

US10584335B2 · kind B2 · utility

Assignee

• Sirna Therapeutics, Inc. · US

Inventors

• Lee Lim · San Francisco, US
• Guillaume Chorn · Hong Kong, CN
• Aarron T. Willingham · San Francisco, US
• Lihong Zhao · Walnut Creek, US

Key dates

• Filing date: Nov 10, 2017
• Grant date: Mar 10, 2020
• Expiry date: Nov 10, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/3515
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

The instant disclosure features single-stranded RNA molecules comprising one or more internal, non-nucleotide spacers. A non-nucleotide spacer covalently links nucleotide portions of the molecule. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting gene expression via an RNA interference mechanism and, thus, represent single-stranded RNAi agents. The single-stranded RNAi molecules can be used in methods for a variety of therapeutic, diagnostic, target validation, genomic discovery, genetic engineering, and pharmacogenomic applications.