vandefitemcel

Vandefitemcel (SB623) is an investigational allogeneic cell therapy developed by SanBio for the treatment of chronic neurological motor deficits. It consists of bone marrow-derived mesenchymal stem cells (MSCs) from healthy donors that have been genetically modified with a plasmid encoding the human Notch1 intracellular domain (NICD). This modification is intended to enhance the cells' ability to promote neural regeneration without permanent integration into the host tissue. The therapy is administered via stereotactic intracranial implantation directly into the area surrounding the brain injury (e.g., the peri-infarct zone in stroke or the site of a traumatic brain injury). Once implanted, the cells act as "bioreactors," secreting a variety of trophic factors—such as vascular endothelial growth factor (VEGF) and hepatocyte growth factor (HGF)—that stimulate endogenous repair mechanisms, including angiogenesis, neurogenesis, and synaptic plasticity. While vandefitemcel received conditional and time-limited approval in Japan (under the brand name AKUUGO) for the treatment of traumatic brain injury in 2024, its Phase 2b clinical trial (ACTIsS) for chronic ischemic stroke did not meet its primary efficacy endpoint.

Brand names

AKUUGO

Other names

modified mesenchymal stem cellsallogeneic bone marrow-derived mesenchymal stem cellsmodified stromal cells