Research programme: gene therapies - Genethon/ViroMed

Drug Profile

Research programme: gene therapies - Genethon/ViroMed

Alternative Names: AAV vector containing modified U7 snRNA gene for Duchenne muscular dystrophy - Genethon; AAV vector transferred UGT1A1 gene - Genethon; Gene therapy for Crigler-Najjar syndrome - Genethon; Gene therapy for Duchenne muscular dystrophy - Genethon; Gene therapy for Leber's hereditary optic neuropathy (LHON) - Genethon; Gene therapy for myotubular myopathy - Genethon/Wake Forest University School of Medicine; SMN1 gene therapy for spinal muscular atrophy - Genethon; VM 101 - ViroMed; VM 102; VM 103; VM 104; VM 105

Latest Information Update: 22 Jun 2015

Price : $50

At a glance

  • Originator ViroMed Co Ltd
  • Developer Genethon; ViroMed; Wake Forest University School of Medicine
  • Class Gene therapies
  • Mechanism of Action Dystrophin expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Congenital structural myopathies; Duchenne muscular dystrophy; Eye disorders; Immunological disorders; Liver disorders; Metabolic disorders; Musculoskeletal disorders; Spinal muscular atrophy

Most Recent Events

  • 07 May 2015 Pharmacodynamics data from preclinical studies in Liver disorders (Crigler-Najjar syndrome) released by Genethon
  • 17 Jul 2013 This research collaboration is still ongoing
  • 24 Jun 2013 Preclinical trials in Congenital structural myopathies in France (Parenteral) prior to June 2013
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