Drug Profile

AMP 101

Alternative Names: AAV-D7; AAV-Dok7; AAV-Dok7 gene therapy; AMP-101

Latest Information Update: 04 Jun 2023

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At a glance

Originator University of Tokyo
Developer Amplo Biotechnology
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Congenital myasthenic syndromes
New Molecular Entity No

Highest Development Phases

Preclinical Congenital myasthenic syndromes; Neuromuscular disorders

Most Recent Events

29 May 2023 Amplo Biotechnology plans to begin clinical trials for Congenital myasthenic syndromes in 2024
06 Oct 2021 Amplo Biotechnology receives fast-track phase I/II SBIR grant from the National Institutes of Health (NIH) for AMP 101 development in Congenital myasthenic syndromes
06 Oct 2021 Preclinical trials in Congenital myasthenic syndromes in USA (IV)

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