Amlogenyx

Amlogenyx is dedicated to the development of a novel gene therapy strategy to reduce amyloid accumulation in the brain in order to prevent and treat diseases like Alzheimer’s disease. We are developing a novel protease therapy shown to reduce amyloid (Aβ42) accumulation in preclinical models of Alzheimer’s disease. Amyloid accumulation has been shown to predict progression of Alzheimer's disease, and treatment with monoclonal antibodies to reduce amyloid has been shown to potentially slow the disease but not stop or reverse it. There is a clear need for better treatment options. Dr. Alessandra d’Azzo, Ph.D., at St. Jude Children’s Research Hospital in Memphis identified a more potent method of degrading amyloid within neuronal cells that relies on the protective protein cathepsin A (PPCA)/neuraminidase (neu1) complex. Ultragenyx studied the effects of PPCA in vitro and showed it could potently degrade amyloid in various oligomeric and higher forms. Ultragenyx then worked with Dr. D'Azzo to develop an AAV9 gene therapy that can deliver supplemental PPCA enzyme to the brain and has shown a substantial reduction in Aβ42 accumulation in murine models of Alzheimer’s disease that exceeds that observed with monoclonal antibodies. Amlogenyx is a subsidiary of Ultragenyx.