Compositions and methods of treating facioscapulohumeral muscular dystrophy

IL311452BActive Publication Date: 2026-07-01AVIDITY BIOSCI INC
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Patent Information

Application Number
IL311452
Authority / Receiving Office
IL · IL
Patent Type
Patents
Current Assignee / Owner
Priority Date
2021-09-16
Filing Date
2022-09-15
Publication Date
2026-07-01
Estimated Expiration
2042-09-15

AI Technical Summary

Technical Problem

Facioscapulohumeral muscular dystrophy (FSHD) lacks effective treatments, primarily due to the aberrant expression of the DUX4 gene leading to inappropriate protein presence, causing progressive skeletal muscle loss and weakness without approved therapeutic options.

Method used

A polynucleic acid molecule conjugate is developed, comprising an antibody or antigen binding fragment conjugated to a polynucleic acid that hybridizes to the DUX4 target sequence, mediating RNA interference to reduce DUX4 mRNA expression, thereby addressing the root cause of muscle pathology.

Benefits of technology

The polynucleic acid conjugate effectively reduces DUX4 mRNA transcript levels by at least 50% in muscle cells, modulating muscle atrophy and dystrophy symptoms, providing a potential disease-modifying therapeutic approach for FSHD.

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Abstract

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating facioscapulohumeral muscular dystrophy (FSHD).
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