Muscle targeting complexes and uses thereof for treating myotonic dystrophy

JP2025067909A5Active Publication Date: 2025-10-17DYNE THERAPEUTICS INC
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Patent Information

Application Number
JP2025004577
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2019-06-10
Filing Date
2025-01-14
Publication Date
2025-10-17
Estimated Expiration
2039-08-02

AI Technical Summary

Technical Problem

Current treatments for myotonic dystrophy type 1 (DM1) are limited, and there is no effective method to specifically target and deliver molecular payloads to muscle cells to inhibit the expression or activity of the disease-causing DMPK allele.

Method used

Development of muscle-targeting complexes that include a muscle-targeting agent, such as a muscle-targeting antibody, covalently linked to a molecular payload. These complexes specifically bind to receptors on the surface of muscle cells, facilitating receptor-mediated internalization and targeted delivery of the molecular payload to the muscle cells.

Benefits of technology

The muscle-targeting complexes effectively deliver molecular payloads to muscle cells, inhibiting the expression or activity of the DMPK allele, thereby providing a potential therapeutic approach for treating myotonic dystrophy type 1.

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Abstract

To provide complexes comprising a muscle-targeting agent covalently linked to a molecular payload.SOLUTION: In some embodiments, a 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.SELECTED DRAWING: None
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