Amplo Biotechnology

Amplo is a late-stage preclinical AAV gene therapy company focused on developing treatments for congenital myasthenic syndromes and neuromuscular junction disorders. Amplo's lead program, AAV-Dok7, was developed by a team led by Professor Yuji Yamanashi of the Institute of Medical Science at the University of Tokyo, and is applicable to various rare and severe diseases. The AAV-Dok7 gene therapy targets defects in the formation of neuromuscular junctions. Administered via a single intravascular injection, the therapy increases the production of Dok7 and activates molecular events that lead to the formation of neuromuscular junctions.