Project Grant R41NS134480

This Project Grant awarded by the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $339,999 to Couragene, Inc. to develop a novel, chemically modified ribonucleoprotein (CRNP) complex for the delivery of CRISPR-mediated gene editing to the brain for the treatment of Angelman syndrome. The project aims to optimize the CRNP formulation to achieve over 70% editing efficiency in primary Angelman syndrome neuronal cells, and then characterize the leading CRNP formulation to determine its genome editing delivery efficiency and safety in Angelman syndrome reporter mice, with a goal of achieving brain-wide genome editing with over 50% neuronal editing efficiency through a single intrathecal injection. The successful completion of this work will build a foundation for further preclinical evaluation and development of a non-viral genome editing therapy for Angelman syndrome using Couragene's CRNP technology.