Drug Profile


Alternative Names: AAV1-gamma-sarcoglycan gene therapy - Genethon; AAV1-gamma-sarcoglycan vector injection - Genethon; Adeno-associated viral 1 vector-mediated gamma-sarcoglycan gene therapy - Genethon; Adeno-associated viral vector containing the human gamma-sarcoglycan gene - Genethon; Adeno-associated virus 1-gamma-sarcoglycan gene therapy - Genethon; Gamma sarcoglycanopathy gene therapy - Genethon

Latest Information Update: 25 Feb 2016

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At a glance

  • Originator ViroMed Co Ltd
  • Developer Genethon
  • 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 - Sarcoglycanopathies
  • New Molecular Entity No

Highest Development Phases

  • Phase I Sarcoglycanopathies

Most Recent Events

  • 25 Feb 2016 This programme is still active
  • 17 Jul 2013 Preclinical trials in Sarcoglycanopathies (gamma sarcoglycanopathy) in France (IV)
  • 12 Jan 2012 Adverse events and pharmacodynamics data from a phase I trial released by Genethon
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