Gene therapy for diseases of the neuromuscular junction
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
President
Program Director
Our Science
ASSET
tARGET
AMP-101
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.
AMP-201
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.
STATUS
10-12 Months from clinical trial application
Lead candidate selection
The
way
01
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.
02
Capital efficient
We are challenging the traditional drug development paradigm so that we can serve rare and ultra-rare patient populations.
03
Focused
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...
In the News
Webinar - 2024
Amplo’s World CMS Patient Webinar to share the progress of AMP-101. Follow the link to register:
Press - October 2021
Getting in touch
commdev@amplobiotechnology.com
©2023 by Amplo Biotechnology