Oligonucleotide compositions and methods of use thereof

US12391942B2 · kind B2 · utility

Assignee

• WAVE LIFE SCIENCES LTD. · SG

Inventors

• Jason Jingxin Zhang · Walpole, US
• Chandra Vargeese · Schwenksville, US
• Naoki Iwamoto · Watertown, US
• Chikdu Shakti Shivalila · Woburn, US
• Nayantara Kothari · Newton, US
• Ann Fiegen Durbin · Arlington, US
• Selvi Ramasamy · Wayland, US
• Pachamuthu Kandasamy · Cambridge, US
• Jayakanthan Kumarasamy · Arlington, US
• Gopal Reddy Bommineni · Port Jefferson, US
• Subramanian Marappan · Acton, US
• Sethumadhavan Divakaramenon · Lexington, US
• David Butler · Medford, US
• Genliang Lu · Winchester, US
• Hailin Yang · Riverdale, US
• Mamoru Shimizu · Uruma, JP
• Prashant Monian · Arlington, US

Key dates

• Filing date: May 10, 2019
• Grant date: Aug 19, 2025
• Expiry date: Jan 30, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2330/30
• WIPO field: Organic fine chemistry
• WIPO sector: Chemistry

Abstract

Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51 or exon 53, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.