bone marrow-derived mesenchymal stromal cells (Emory University)

This investigational cell therapy consists of allogeneic, bone marrow-derived mesenchymal stromal cells (MSCs) developed by Emory University for the treatment of severe Osteogenesis Imperfecta (OI) Type 3. The therapy is currently being evaluated in a Phase 1/2 clinical trial (NCT05559801) targeting children aged 3 to 10 years. The MSCs are administered intravenously and are intended to improve linear growth rates and bone health parameters, which are severely compromised in this patient population. The proposed mechanism of action involves the cells' ability to home to sites of bone injury or active remodeling, where they may differentiate into functional osteoblasts and secrete paracrine factors that modulate the bone microenvironment, stimulate endogenous bone formation, and potentially produce healthy collagen to compensate for the genetic defects characteristic of OI.

Other names

allogeneic bone marrow-derived mesenchymal stromal cellsMSCs