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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, 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.
Amplo is led by experienced AAV entrepreneurs:
Patricio's experience includes Directing the technology transfer office of the Royal Melbourne Hospital, Senior Manager in  M&A and Licensing at CSL Behring, managing global research projects for the Movember Foundation, CSO at Myostin Therapeutics, working as an analyst at the Venture Capital firm Brandon Capital Partners and guest scientist at the Department of Neuropisiology of Uppsala University.
An adviser to Save Our Sons Duchenne Australia, Duchenne Alliance US, PNET Cancer Foundation and private consultant to Milo Biotechnology.  He conducted his PhD in AAV Gene Therapy at the Baker Research Institute and has an Executive MBA from the Melbourne Business School at the University of Melbourne
Al Hawkins is a gene therapy entrepreneur and investor.  He was co-founder and Chairman of AAV gene therapy company Abeona Therapeutics (NASDAQ: ABEO), which has translated two therapies into clinical studies for rare lysosomal storage diseases. He is also Co-Founder and CEO of Milo Biotechnology; Milo is a mid-clinical stage AAV gene therapy company that has established proof-of-concept in two muscular dystrophy indications.
Hawkins formerly was VP of Business Development at therapeutics accelerator BioMotiv and Director of the Boston University Venture Fund.  He received an SM in Health Sciences and Technology from the Harvard-MIT Institute of Health Sciences, an MBA from UW-Madison and a BA from Emory University.
AMP-101 gene targets neuromuscular junction formation and stability.  Delivered once, AMP-101 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 and in Amplo's pharmacology studies, AMP-101 cures mice lacking Dok7 protein.
AMP-101 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.