2nd Edition of Cell & Gene Therapy World Conference 2026

Abstract

Mesenchymal stromal cells (MSCs) remain a cornerstone of regenerative medicine, yet their heterogeneous composition frequently results in inconsistent clinical outcomes and therapeutic limitations. Within MSC populations resides a rare pluripotent subpopulation (1–3%) of multilineage-differentiating stress-enduring (Muse) cells, identified by SSEA-3 positivity and expression of OCT4, SOX2, and NANOG. Naïve perinatal Muse cells demonstrate superior tissue-repair mechanisms compared to conventional bone-marrow or adipose MSCs. They exhibit preferential homing to injured tissues via the sphingosine-1-phosphate (S1P)–S1PR2 axis, spontaneous differentiation into appropriate functional cell types (e.g., neurons in stroke models, hepatocytes post-hepatectomy, dermal/epidermal cells in diabetic ulcers), and robust paracrine effects without teratoma formation. Liquid chromatography–tandem mass spectrometry (LC-MS/MS) secretome profiling reveals 56 proteins exclusive to Muse cells, plus unique upstream regulators absent in standard MSCs: cytokines (CRH, LIF), growth factors (BMP4, FGF18), transcription factors (DLX4, GATA3, AIRE, FHL2), and miRNAs (miR-146, let-7, miR-182, miR-140). These confer enhanced immunomodulation, angiogenesis, anti-apoptosis, anti-scarring, and targeted regeneration. Intravenous delivery of Muse cells or their exosomes has produced promising preclinical results across osteoarthritis, ischemic stroke, myocardial infarction, chronic wounds, liver fibrosis, and age-related degenerative conditions. Perinatal sourcing further improves scalability, consistency, and manufacturing feasibility. ANM Health LLC is advancing perinatal Muse cell-derived exosomes as an off-the-shelf, cell-free platform that directly addresses key translational challenges of traditional MSC therapies — product variability, limited engraftment, and modest efficacy — while offering safer, more potent, and more reproducible clinical outcomes.