Gene therapeutics to strengthen muscle and fight disease
AAV vectors are the holy grail of gene therapy. These viral tools introduce "payload genes" into different tissues often with great precision. They do not cause disease and have a proven safety record in clinical trials. Thus, their use has the potential to provide safe and long-term relief for genetic and/or chronic diseases.
AAVogen is developing a gene therapy for muscle wasting diseases, especially inclusion body myositis, Duchenne muscular dystrophy and cancer cachexia. In pre-clinical studies, AVGN7 stabilizes muscle structure and increases muscle mass, strength and exercise capacity. It also prevents muscle wasting.