Muscle targeting complexes and uses thereof for treating dystrophinopathies

US11633496B2 · kind B2 · utility

Assignee

• Dyne Therapeutics, Inc. · US

Inventors

• Romesh R. Subramanian · Framingham, US
• Mohammed T. Qatanani · Waltham, US
• Timothy Weeden · Waltham, US

Key dates

• Filing date: Sep 8, 2022
• Grant date: Apr 25, 2023
• Expiry date: Sep 8, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/33
• 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 promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.