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Research programme: gene therapies - Genethon/Helixmith

Drug Profile

Research programme: gene therapies - Genethon/Helixmith

Alternative Names: AAV vector containing modified U7 snRNA gene for Duchenne muscular dystrophy - Genethon; Gene therapy for Duchenne muscular dystrophy - Genethon; SMN1 gene therapy for spinal muscular atrophy - Genethon; VM 101 - Helixmith; VM 102; VM 103; VM 104; VM 105

Latest Information Update: 08 Aug 2019

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At a glance

  • Originator ViroMed Co Ltd
  • Developer Genethon; Helixmith
  • 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 Duchenne muscular dystrophy; Immunological disorders; Metabolic disorders; Musculoskeletal disorders; Spinal muscular atrophy

Most Recent Events

  • 29 Mar 2019 ViroMed plans a phase I trial for gene therapy candidates, by 2021
  • 03 Jul 2018 Preclinical development is ongoing in France and South Korea (Genethon pipeline, ViroMed webiste, July 2018)
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in France (Parenteral)
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