Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups

US9469664B2 · kind B2 · utility

Assignee

• Sarepta Therapeutics, Inc. · US

Inventors

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

Key dates

• Filing date: Jun 6, 2014
• Grant date: Oct 18, 2016
• Expiry date: Jun 6, 2034

Classification

• Technology area (CPC Y): Emerging Cross-Sectional Technologies
• CPC primary: Y02P20/55
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Oligonucleotide analogs 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.