Nucleic acid aptamers recognizing the extra cellular domain of alpha7 / beta1 integrin dimers and uses thereof

Aptamers targeting alpha7/beta1 integrin dimers provide selective and efficient delivery of therapeutic molecules to skeletal muscle fibers and satellite cells, addressing the challenges of current delivery systems and demonstrating therapeutic and diagnostic efficacy.

WO2025210585A2 Publication Date: 2025-10-09UNIV CATTOLICA DEL SACRO CUORE +4
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Patent Information

Application Number
PCT/IB2025/053587
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-04-05
Filing Date
2025-04-04
Publication Date
2025-10-09

AI Technical Summary

Technical Problem

Current delivery systems for therapeutic molecules to skeletal muscle, particularly targeting satellite cells, face challenges in selective and efficient uptake due to the vast extension of the tissue and lack of precise targeting, especially when administered systemically.

Method used

Development of aptamers that selectively bind to the alpha7/beta1 integrin dimer, a surface protein highly enriched in skeletal muscle fibers and satellite cells, allowing efficient delivery of therapeutic molecules, including nanoformulations, even to inaccessible muscles like the diaphragm, and targeting aberrantly expressing tumor cells.

Benefits of technology

The aptamers demonstrate high affinity and specificity for alpha7/beta1 integrin dimers, enabling efficient delivery to skeletal muscle fibers and satellite cells, stimulating muscle regeneration and showing therapeutic potential in animal models, while also being useful for diagnostic and research applications.

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Abstract

The present invention relates to aptamers that specifically bind to the extra-cellular domain of alpha7 / beta1 integrin dimers, conjugates or particles comprising said aptamers, in muscles cells such as skeletal muscle fibres and satellite cells, or in cells aberrantly expressing said domain e.g. tumour cells such as rhabdomyosarcoma (muscle-derived tumours), or glioblastoma cancer stem cells, as well as conjugates comprising said aptamers, therapeutic or diagnostic compositions comprising said conjugates or said aptamers, and their use as a medicament and in diagnostic methods.
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