This federal Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, is supporting the development of a novel epigenetic editing therapy for Rett syndrome. The $698,459 award to Epitor Therapeutics, Inc. will fund research to optimize an ultracompact CRISPR-based epigenetic editor, composed of a catalytically dead Cas (dCas) protein fused to a potent TET demethylase domain. This epigenetic editor is designed for targeted delivery to the brain using adeno-associated viral (AAV) vectors to reactivate the silenced but functional MECP2 gene in Rett syndrome patient neurons. The project aims to assess the editor's DNA targeting ability, specificity, and efficacy in patient-derived cells and a mouse model of Rett syndrome, with the goal of advancing a potential first-in-class epigenetic editing treatment for this devastating neurodevelopmental disorder. The award period runs from September 2024 to February 2026.
Generated 3/4/25, 6:59 AM