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. Amplo’s lead program, 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.
Amplo is led by experienced AAV entrepreneurs:
AAV-Dok7 gene therapy targets defects in neuromuscular junction formation. Delivered once, via intravascular injection, the therapy increases production of Dok7 and activation of molecular events that lead to neuromuscular junction formation.
Amplo’s initial focus is Dok7 congenital myasthenia syndrome (CMS), a disease that causes systemic muscle wasting, difficulty in breathing and swallowing. Dok7 CMS affects approximately 2,000-4,000 patients in the major markets. In preclinical studies conducted at the University of Tokyo, AAV-Dok7 cures mice lacking Dok7 protein.
AAV-Dok7 is also applicable to other diseases in which the neuromuscular junction is compromised. The investigators at the University of Tokyo have demonstrated significant benefits in mouse models of amyotrophic lateral sclerosis (ALS) and Emery-Dreifuss muscular dystrophy.
Sumimasa Arimura et al. Science 2014;345:1505-1508
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