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Gene therapy for diseases of the neuromuscular junction

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How and Who?

We are working with the best, around the globe and around the clock to serve patients in need of our therapies.

Collaborators and supporters

Leadership Team

Al Hawkins


Claudia Canzonetta

Program Director

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Our Science




AAV-Dok7 gene therapy

The Dok-7 protein is central to the development and maintenance of the neuromuscular junction, the critical connection between muscle and nerve. AMP-101 is a muscle-targeted AAV therapy delivering the Dok-7 protein, with initial application in Congenital Myasthenic Syndromes, genetic diseases of the neuromuscular junction. AMP-101’s downstream applications are broad: Emery-Dreyfuss Muscular Dystrophy, ALS, and ageing, to name a few.


AAV-Collagen-Q gene therapy

The Collagen Q (ColQ) protein is critical for the anchoring of acetylcholinesterase, which attaches to the endplates of neuromuscular junctions and is essential for normal skeletal muscle function. AMP-201 is based on targeted AAV expression of ColQ to treat ColQ Congenital Myasthenic Syndrome.

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10-12 Months from clinical trial application

Lead candidate selection



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Removing risks

We are not early adopters. We use known delivery technologies and known manufacturing methods. Let the risk be in the indication, with no distractions.


Capital efficient

We are challenging the traditional drug development paradigm so that we can serve rare and ultra-rare patient populations.



Amplo is dedicated only to developing AAV therapies for diseases of the neuromuscular junction (NMJ), working with the world’s leading NMJ experts.

Let's help the JJs and Max's of the world...

JJ and Max suffer from Dok-7 CMS, they are not giving up, nor we are. We hope to bring them a definitive treatment soon...

Getting in touch


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