©2019 by Amplo Biotechnology

Focused on Cures for the Neuromuscular Junction

 

OVERVIEW

Chronic autoinmune and congenital myasthenia are diseases of the neuromuscular junction with profound health effects including paralysis, breathing and swallowing difficulties. Amplo Biotechnology plans to license and develop multiple, potentially curative, Adeno-Associated Viral (AAV) therapies for the neuromuscular junction with an initial focus on myasthenias.  Amplo’s initial 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 neuromuscular diseases.  

Amplo is led by experienced AAV entrepreneurs:

Al Hawkins

Co-Founder

Patricio Sepulveda

Co-Founder

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 TECHNOLOGY

Amplo Biotechnology’s AAV-Dok7 gene therapy targets defects in neuromuscular junction transmission.  Delivered once via intravascular injection, the therapy increases production of Dok7, a protein that is an activator of the muscle-specific receptor tyrosine kinase MuSK. 

Amplo’s initial focus is congenital Dok7 myasthenia, a rare neuromuscular disease that causes systemic muscle wasting and, eventually, loss of walking ability.  Dok7 myasthenia affects approximately 2,000-4,000 patients in the U.S., EU, Japan and Australia.  Preclinical studies conducted at the University of Tokyo demonstrated that the AAV-Dok7 therapy rescued a mouse model of Dok7 myasthenia.

AAV-Dok7 is also applicable to other diseases in which the neuromuscular junction is compromised.  The investigators at the University of Tokyo also 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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