nVSM

nVSM is an investigational cell-based gene therapy being developed by Nervosave Therapeutics for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD). The therapy utilizes neonatal vascular smooth muscle (nVSM) cells, which are uniquely characterized by their ability to migrate through the circulatory system, exit the vasculature, and fuse with skeletal muscle fibers. In the context of FSHD, these cells are engineered to carry a genetic payload designed to repress the expression of the DUX4 gene. FSHD is caused by the inappropriate expression of the DUX4 protein in muscle cells, leading to progressive muscle weakness and atrophy. By fusing with existing myofibers, the nVSM cells distribute the repressor throughout the muscle tissue, effectively silencing DUX4 and potentially halting or reversing the disease pathology. This systemic delivery approach distinguishes nVSM from other cell therapies that require direct intramuscular injection.

Other names

neonatal Vascular Smooth Muscle cells