Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

US11679161B2 · kind B2 · utility

Assignee

• Dyne Therapeutics, Inc. · US

Inventors

• Romesh R. Subramanian · Framingham, US
• Mohammed T. Qatanani · Waltham, US
• Timothy Weeden · Waltham, US
• Cody A. Desjardins · Waltham, US
• Brendan Quinn · Boston, US
• John Najim · Waltham, US

Key dates

• Filing date: Dec 9, 2022
• Grant date: Jun 20, 2023
• Expiry date: Dec 9, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/32
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.