Muscle targeting complexes and uses thereof for treating myotonic dystrophy

US11911484B2 · 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

Key dates

• Filing date: Mar 18, 2021
• Grant date: Feb 27, 2024
• Expiry date: Mar 18, 2041

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y207/11001
• 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 a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.