haploidentical bone marrow-derived mesenchymal stem cells

This investigational cell therapy consists of haploidentical bone marrow-derived mesenchymal stem cells (BM-MSCs) developed by the Instituto de Investigación Hospital Universitario La Paz for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe genetic skin disorder caused by mutations in the COL7A1 gene, which leads to a deficiency in type VII collagen and subsequent skin fragility and blistering. The therapy involves the systemic intravenous infusion of allogeneic MSCs, which are hypothesized to migrate to damaged skin sites, promote tissue regeneration, and exert immunomodulatory effects to reduce inflammation and fibrosis. A Phase I/II pilot trial (NCT04520022) has evaluated the safety and preliminary efficacy of these cells in pediatric patients, focusing on the restoration of type VII collagen function at the dermal-epidermal junction.

Other names

BM-MSCshaploidentical BM-MSCsallogeneic mesenchymal stromal cells