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AMP 101

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Drug Profile

AMP 101

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

Latest Information Update: 28 Nov 2025

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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

  • No development reported Congenital myasthenic syndromes; Neuromuscular disorders

Most Recent Events

  • 28 Nov 2025 No recent reports of development identified for preclinical development in Congenital-myasthenic-syndromes in USA (IV, Injection)
  • 28 Nov 2025 No recent reports of development identified for preclinical development in Neuromuscular-disorders in USA (IV, Injection)
  • 29 May 2023 Amplo Biotechnology plans to begin clinical trials for Congenital myasthenic syndromes in 2024

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