Exploring a New Approach to Alzheimer’s – Through Gene Therapy
Alzheimer’s disease remains a devastating and progressive condition, despite advances in treatments that target amyloid. At Amlogenyx, we are developing AM805, an investigational one-time gene therapy designed to address amyloid accumulation through a fundamentally different approach.
By delivering a naturally occurring enzyme that breaks down amyloid in the brain, AM805 is designed to reduce amyloid accumulation. This approach has the potential to address important limitations of current therapies and support a new treatment paradigm for people living with Alzheimer’s disease.
Vision
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.
About the science
A novel, more potent approach to treating Alzheimer’s Disease
A growing body of research is revealing lysosomal dysfunction as an important feature of Alzheimer’s disease and other neurodegenerative conditions, linking impaired cellular clearance to the accumulation of toxic proteins.
Our research focuses on protective protein cathepsin A (PPCA), a lysosomal protease capable of breaking down Aβ amyloid. In preclinical models of Alzheimer’s disease, adeno-associated virus (AAV) gene therapy that increases PPCA levels in the brain reduced both intracellular amyloid and extracellular plaque, supporting the potential of harnessing lysosomal biology as a new therapeutic strategy.
We believe the lysosome represents an underexplored opportunity in neurodegenerative disease. By targeting lysosomal biology, we aim to advance a fundamentally different approach to treatment, with the potential for lasting benefit from a single administration.
Learn more about our science here
