Muscle-targeting complex and its use for treating myotonic dystrophy

Oligonucleotides conjugated with muscle-targeting agents specifically degrade DMPK RNA in muscle and CNS cells, addressing the lack of effective DM1 treatments by reducing mRNA levels and correcting splicing defects.

JP2026086413APending Publication Date: 2026-05-26DYNE THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
DYNE THERAPEUTICS INC
Filing Date
2026-01-09
Publication Date
2026-05-26

AI Technical Summary

Technical Problem

Current treatments for myotonic dystrophy type 1 (DM1) are ineffective, and there is a need for targeted therapies to reduce levels of toxic DMPK RNA and inhibit mutant DMPK expression in muscle and nervous system cells.

Method used

Development of oligonucleotides designed for RNAeH-mediated degradation of DMPK RNA, conjugated with muscle-targeting agents like anti-transferrin receptor 1 antibodies, to deliver the oligonucleotides specifically to muscle and CNS cells, reducing DMPK expression through receptor-mediated endocytosis and endosomal release.

Benefits of technology

The oligonucleotide complexes effectively reduce DMPK mRNA levels by at least 30% and correct splicing defects, leading to a significant reduction in nuclear foci and phenotypic improvements in muscle cells and animal models.

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Abstract

This application relates to oligonucleotides designed to target DMPK RNA (e.g., antisense oligonucleotides such as gapmer), targeted conjugates for delivering oligonucleotides to cells (e.g., muscle cells), and their uses, particularly in relation to the treatment of diseases. [Solution] In some embodiments, the muscle targeting agent specifically binds to internalized cell surface receptors on muscle cells. In some embodiments, the molecular payload inhibits the expression or activity of DMPK.
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