Amplo Biotechnology 

Patricio Sepulveda, Ph.D., CEO 

Oct. 11 | 4:45pm | Oxford Biomedica Ballroom

Towson, MD


In-person Presentation 

Amplo Biotechnology is a late preclinical stage company developing AAV therapies to treat diseases of the neuromuscular junction, the connection between muscle and nerve.  Amplo’s lead asset, AMP-101, uses a muscle specific AAV construct expressing the Dok7 protein.  Patients who do not produce Dok7 develop Dok7 Congenital Myasthenic Syndrome, a severe rare disease of the neuromuscular junction. AMP-101 is able to cure the Dok7 mouse model with a dose that is ¼ the effective dose in other systemic muscle targeted AAV programs.  AMP-101 is further applicable to other forms of congenital myasthenic syndromes, Emery Dreifuss muscular dystrophy, ALS and sarcopenia, all for which there is animal data supporting AMP-101’s ability to stabilize the neuromuscular junction and improve muscle function. Amplo is currently conducting a dose finding pharmacology study and GLP tox studies on AMP-101, with the aim of a 2023 IND submission.


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