Pre-Clinical Development of EDN-OL1 for Alzheimer's Disease

National Center For Advancing Translational Sciences

Investigators

Abstract

More than 5 million Americans now have Alzheimers disease (AD), the sixth leading cause of death in the United States, and no disease-modifying drug is available. EDN-OL1, a patented antisense phosphorothioate oligonucleotide, crosses the blood-brain barrier after intravenous dosing and reduces production of amyloid precursor protein (APP) and, consequently, amyloid proteins in the brains of mouse models of AD. The clinical benefit to AD patients may best be anticipated as a decrease in the rate of cognitive decline. Edunn requested program assistance to carry out specific tasks for pre-clinical development of EDN-OL1 to support submission of an Investigational New Drug (IND) application to enable clinical trials for the Alzheimers indication, ultimately leading to FDA approval for treatment of AD patients. The lab has made progress towards the completion of the following studies: - Synthesis of Good Manufacturing Practice (GMP) and non-GMP material - Formulation development