Description
2BAct (eIF2B activator 2BAct) is a novel, highly selective, CNS-permeable small molecule eIF2B activator with EC50 of 33nM in cell-based reporter assays; 2BAct normalized body weight gain and prevented motor deficits in male R191H mice, prevented myelin loss and reactive gliosis in the brain and spinal cord of R191H mice; also prevented ISR induction in the cerebellum and spinal cord of Eif2b5R132H/R132H mice, prevented all measures of pathology and normalizes the transcriptome and proteome of Vanishing White Matter (VWM) mice