ASP2246

ASP2246 is an investigational mRNA-based therapy developed by Astellas Pharma Inc for the treatment of motor dysfunction in patients with chronic ischemic stroke. The drug consists of mRNA encoding the transcription factor Neurogenic Differentiation 1 (NeuroD1) encapsulated in lipid nanoparticles (mRNA-LNPs). Administered via direct stereotactic intracerebral parenchymal infusion, the therapy aims to achieve in vivo cellular reprogramming of resident astrocytes into functional neurons within the infarcted area (specifically the supratentorial perforator area). By facilitating neural regeneration and the reconstruction of neural circuits, ASP2246 is intended to improve motor outcomes in stroke survivors who have persistent disability and have progressed beyond the window of acute intervention.