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allogeneic mesoangioblasts
- Development stage
- Unknown
- Lead developer
- Fondazione Centro San Raffaele
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intra-arterial
01
Overview
Allogeneic mesoangioblasts is an investigational cell therapy consisting of HLA-matched, vessel-associated myogenic progenitor/stem cells derived from healthy family donors. Developed by the Fondazione Centro S. Raffaele del Monte Tabor (IRCCS San Raffaele Scientific Institute), this therapy is designed to treat Duchenne muscular dystrophy (DMD). The cells are administered via repeated intra-arterial delivery to target skeletal muscle regeneration and restore dystrophin expression. A Phase I/IIa clinical trial (EudraCT No. 2011-000176-33, protocol DMD03) evaluated the safety, engraftment, and efficacy of these cells in pediatric patients with DMD under tacrolimus immunosuppression, demonstrating safety but limited functional efficacy.
- Other names
- HLA-identical allogeneic mesoangioblastsallogeneic mesoangioblasts
02
Targets
DMD (Dystrophin)
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