Muscle-Targeted Gene Therapy for Disease-Modifying Treatment of Alzheimer's Disease

Abstract

Alzheimer's disease is a progressive neurodegenerative disorder characterized by memory loss, cognitive decline, and loss of motor function, with no therapies that reliably slow or halt disease progression. Most current treatments focus on amyloid-beta plaque reduction through monoclonal antibodies or small-molecule inhibitors, yet these approaches have shown limited clinical benefit, high development costs, and significant safety and delivery challenges. Many patients experience minimal cognitive improvement while facing risks such as inflammation, imaging abnormalities, or poor tolerability. Critically, these strategies fail to address broader neurodegenerative processes such as impaired neurogenesis, synaptic dysfunction, and systemic contributors to brain health, leaving a substantial unmet need for disease-modifying therapies.

Researchers at Florida Atlantic University have developed a novel muscle-targeted gene therapy that harnesses the muscle–brain signaling axis to deliver disease-modifying benefits for Alzheimer's disease without direct brain intervention. The innovation uses an adeno-associated viral (AAV) vector to drive muscle-specific expression of Cathepsin B, a protein associated with neurogenesis, synaptic plasticity, and memory function. By leveraging peripheral expression, the therapy avoids the delivery, safety, and scalability limitations of brain-targeted amyloid therapies while producing systemic effects that improve cognition and motor function. The technology has demonstrated compelling efficacy in established Alzheimer's disease animal models, with ongoing opportunities for translational development, IND-enabling studies, and partnership-led advancement toward clinical evaluation.

FAU seeks to advance this innovation into the marketplace through licensing or development partnerships.

Benefit

Market Application

Publications

Muscle Cathepsin B Treatment Improves Behavioral and Neurogenic Deficits in a Mouse Model of Alzheimer's Disease