Extracellular vesicles for therapy

Intravenous administration of bone marrow-derived mesenchymal stromal cell extracellular vesicles effectively addresses VIDD and CIM by restoring muscle fiber size and function, offering a promising treatment for muscle wasting induced by mechanical ventilation and other causes.

WO2025250774A1 Publication Date: 2025-12-04LEKSUM LLC
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Patent Information

Application Number
PCT/US2025/031391
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-05-31
Filing Date
2025-05-29
Publication Date
2025-12-04

AI Technical Summary

Technical Problem

Current treatments for ventilator-induced diaphragm dysfunction (VIDD) and critical illness myopathy (CIM) are ineffective, and there is a need for therapies to counteract muscle wasting due to prolonged mechanical ventilation, immobilization, drug side effects, or age-related muscle loss.

Method used

Intravenous administration of extracellular vesicles derived from bone marrow-derived mesenchymal stromal cells to mitigate muscle wasting and diaphragm dysfunction, using a therapeutic amount of 200,000 to 1,000,000 vesicles per kg of subject body mass, administered via various methods including intravenous, subcutaneous, or inhalation.

Benefits of technology

The extracellular vesicles significantly restore diaphragm muscle fiber size and function, reducing muscle atrophy and improving lung health, as demonstrated by proteomic and metabolomic analyses, potentially shortening weaning from mechanical ventilation.

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Abstract

This disclosure provides a secretion centric therapy to overcome muscle wasting. Provided are mesenchymal stem cell secretion-based therapies to overcome muscle wasting due to: old age; as a consequence of immobilization (e.g., mobility restricted or paralytics administered); due to use of ventilators (e.g., in an ICU or long-term immobilization); in microgravity during long space flights; or, resulting from drug side effects (e.g., the use of weight loss drugs, chemotherapies, etc.). Uris disclosure further provides methods and compositions for modulating, including regulating or altering, the structure and function of diaphragm muscle cells and / or diaphragm muscle fibers in particular, and skeletal muscle in general. In certain embodiments extracellular vesicles are applied to patients affected by muscle wasting.
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