Functionally-modified oligonucleotides and subunits thereof

US10344281B2 · kind B2 · utility

Assignee

• Sarepta Therapeutics, Inc. · US

Inventors

• Gunnar J. Hanson · Cambridge, US
• Dwight D. Weller · Corvallis, US
• Bao Zhong Cai · Corvallis, US
• Ming Zhou · Coppell, US

Key dates

• Filing date: Sep 11, 2017
• Grant date: Jul 9, 2019
• Expiry date: Sep 11, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/353
• WIPO field: Organic fine chemistry
• WIPO sector: Chemistry

Abstract

Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.