Amplo is a late preclinical AAV gene therapy company focused on developing therapies for congenital myasthenic syndromes and conditions of the neuromuscular junction. Diseases of the neuromuscular junction have profound health effects including paralysis, breathing and swallowing difficulties. Amplo Biotechnology plans to develop multiple, potentially curative, Adeno-Associated Viral (AAV) therapies for the neuromuscular junction with an initial focus on congenital myasthenic syndromes. Amplos lead program, AMP-101 (AAV-Dok7), was developed by Professor Yuji Yamanashi's group of the Institute of Medical Science at the University of Tokyo, and is applicable to multiple rare, severe diseases. At the time of start-up, Aomplo management licensed AAV-DOK7 from the University of Tokyo
