Drug intelligence / Profile preview

autologous skeletal myoblasts

Development stage
Phase 2
Lead developer
Bioheart
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intramuscular (direct Injection Into Affected Muscle Or Myocardium), Intracardiac (direct Injection Into Cardiac Tissue For Heart Indications)
01

Overview

rejuvenative Autologous skeletal myoblasts** are muscle progenitor cells derived from a patient's own skeletal muscle tissue, typically through biopsy and in vitro expansion. They originate from satellite cells and possess high proliferative capacity, resistance to ischemia, and a committed myogenic lineage. In cell therapy protocols, these myoblasts are harvested, cultured under GMP conditions, and then transplanted back into the same patient to promote tissue regeneration in degenerative muscle conditions or for cardiac repair. Their therapeutic mechanisms include myogenic differentiation, secretion of paracrine factors (supporting angiogenesis and matrix preservation), and scaffolding effects in damaged myocardium. Clinical applications explored include ischemic and nonischemic cardiomyopathy, Duchenne muscular dystrophy, fecal incontinence, myogenic ptosis, and urinary incontinence. While animal studies showed robust engraftment and tissue regeneration, clinical trials for cardiac indications demonstrated mixed results, with improvements in ejection fraction in some studies but lack of integration into host cardiac tissue and variable impact on contractility[1][2][3][4]. Manufacture uses ex vivo cell expansion of skeletal muscle biopsies using defined, often animal-free media; cells are released for patient use after passing viability, sterility, and functional criteria[2][3]. Immunosuppression is generally not required due to autologous use. Autologous skeletal myoblasts are not a single pharmaceutical formulation or commercial drug, but a **cell therapy product** individually manufactured for each patient.

Other names
autologous myoblastsautologous muscle-derived cells

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